query: first clinical gene therapy was done for the treatment of

pos: Gene therapy case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980. Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified and even if he is correct, it's unlikely it produced any significant beneficial effects treating beta-thalassemia. After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990,

neg: Gene therapy when Ashi DeSilva was treated for ADA-SCID. The first somatic treatment that produced a permanent genetic change was performed in 1993. Gene therapy is a way to fix a genetic problem at its source. The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations. The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a "vector", which carries the molecule inside cells. Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although ,

neg: Choroideremia trials for both choroideremia and Leber's congenital amaurosis (LCA) have produced somewhat promising results. Clinical trials of gene therapy for patients with LCA began in 2008 at three different sites. In general, these studies found the therapy to be safe, somewhat effective, and promising as a future treatment for similar retinal diseases. In 2011, the first gene therapy treatment for choroideremia was administered. The surgery was performed by Robert MacLaren, Professor of Ophthalmology at the University of Oxford and leader of the Clinical Ophthalmology Research Group at the Nuffield Laboratory of Ophthalmology (NLO). In the study, 2 doses of the

neg: Cancer research Cancer research Cancer research is research into cancer to identify causes and develop strategies for prevention, diagnosis, treatment, and cure. Cancer research ranges from epidemiology, molecular bioscience to the performance of clinical trials to evaluate and compare applications of the various cancer treatments. These applications include surgery, radiation therapy, chemotherapy, hormone therapy, immunotherapy and combined treatment modalities such as chemo-radiotherapy. Starting in the mid-1990s, the emphasis in clinical cancer research shifted towards therapies derived from biotechnology research, such as cancer immunotherapy and gene therapy. Cancer research is done in academia, research institutes, and corporate environments, and is largely government funded.

neg: Enzyme replacement therapy from an adenosine deaminase deficiency (ADA-SCID). Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation. ERT was developed in 1964 by Christian de Duve and Roscoe Brady. Leading work was done on this subject at the Department of Physiology at the University of Alberta by Mark J. Poznansky and Damyanti Bhardwaj, where a model for enzyme therapy was developed using rats. ERT was not used in clinical practice until 1991, after the FDA gave orphan drug approval for the treatment of Gaucher disease with Alglucerase. ERTs were

neg: Gene therapy 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results. Clinical trials of gene therapy for sickle cell disease were started in 2014. There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease. In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease. In March

neg: Gene therapy gene of the patient's blood cells was replaced by the functional variant. Ashanti’s immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary. Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993). The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocolno.1602 November

neg: Gene therapy and the original patient had remained tumor-free. Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction. In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF. Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF. The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for

neg: Gene therapy other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient. The protocol for a gene therapy clinical trial must be approved by the NIH's Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial. As with other kinds of drugs, the FDA regulates the quality and safety of gene therapy products and supervises how these products are used clinically. Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects, such as clinical trials,

neg: Treatment of cancer cancer. The goal of this research is to find better ways to treat cancer and help cancer patients. Clinical trials test many types of treatment such as new drugs, new approaches to surgery or radiation therapy, new combinations of treatments, or new methods such as gene therapy. A clinical trial is one of the final stages of a long and careful cancer research process. The search for new treatments begins in the laboratory, where scientists first develop and test new ideas. If an approach seems promising, the next step may be testing a treatment in animals to see how it

neg: Gene therapy "Science" titled "Gene therapy for human genetic disease?" Rogers (1970) was cited for proposing that "exogenous good DNA" be used to replace the defective DNA in those who suffer from genetic defects. In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes. The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson. Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective

neg: Designer baby by the field of gene therapy can potentially be used to create “designer babies”. This can be achieved through the use of IVF to assist in creating a genetically designed baby. Gene therapy is being studied for the treatment of a wide variety of acquired and inherited disorders. Retroviruses, adenoviruses, poxviruses, herpesviruses, and others are being engineered to serve as gene therapy vectors and are being administered to patients in a clinical setting. Some of the other genetic disorders that could potentially be implemented in clinical trials are ADA-SCID, which as stated earlier is severe combined immune deficiency CGD which

neg: Tbx18 transduction effective. Tbx18 transduction Tbx18 transduction is a method of turning on genes in heart muscle cells as a treatment for certain cardiac arrhythmias. Currently this therapy is in the very early stages of experimentation, having only been applied to rodents. Before this treatment can be used in humans, successful tests on larger animals need to be completed followed by human clinical trials. This treatment is one of the many forms of gene therapy that are currently being researched for use in different diseases. Tbx18 gene therapy is aimed at treating a group of arrhythmias known as sick sinus syndrome. In

neg: Tbx18 transduction Tbx18 transduction Tbx18 transduction is a method of turning on genes in heart muscle cells as a treatment for certain cardiac arrhythmias. Currently this therapy is in the very early stages of experimentation, having only been applied to rodents. Before this treatment can be used in humans, successful tests on larger animals need to be completed followed by human clinical trials. This treatment is one of the many forms of gene therapy that are currently being researched for use in different diseases. Tbx18 gene therapy is aimed at treating a group of arrhythmias known as sick sinus syndrome. In a

neg: Genetic engineering (CLL), and Parkinson's disease. In 2012, Alipogene tiparvovec became the first gene therapy treatment to be approved for clinical use. In 2015 a virus was used to insert a healthy gene into the skin cells of a boy suffering from a rare skin disease, epidermolysis bullosa, in order to grow, and then graft healthy skin onto 80 percent of the boy's body which was affected by the illness. Germline gene therapy would result in any change being inheritable, which has raised concerns within the scientific community. In 2015, CRISPR was used to edit the DNA of non-viable human embryos, leading

neg: Gene therapy gene therapy strategy of antisense IGF-I RNA (NIH n˚ 1602) using antisense / triple helix anti-IGF-I approach was registered in 2002 by Wiley gene therapy clinical trial - n˚ 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n˚ LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening

neg: Prostate cancer treatment options is often recommended for managing prostate cancer. Guidelines for treatment for specific clinical situations requires a good estimation of a person's long-term life expectancy. People can also use an 18-item questionnaire to learn whether they have good knowledge and understanding about their treatment options before they choose. Most of those who are newly diagnosed and made a treatment choice can not correctly answer over half of the questions. If radiation therapy is done first, and fails, then radical prostatectomy becomes a very technically challenging surgery and may not be feasible. On the other hand, radiation therapy done after

neg: Leukodystrophy velocities. Next generation multigene sequencing panels for undifferentiated leukodystrophy can now be offered for rapid molecular diagnosis after appropriate genetic counselling. Specific types of leukodystrophies include the following with their respective ICD-10 codes when available: With many different types of leukodystrophies and causes, treatment therapies vary for each type. Many studies and clinical trials are in progress to find treatment and therapies for each of the different leukodystrophies. Stem cell transplants and gene therapy appear to be the most promising in treating all leukodystrophies providing it is done as early as possible. For hypomyelinating leukodystrophies, therapeutic research into cell-based therapies

neg: Gene therapy for epilepsy Gene therapy for epilepsy Gene therapy is being studied for some forms of epilepsy. It relies on viral or non-viral vectors to deliver DNA or RNA to target brain areas where seizures arise, in order to prevent the development of epilepsy or to reduce the frequency and/or severity of seizures. Gene therapy has delivered promising results in early stage clinical trials for other neurological disorders such as Parkinson's disease, raising the hope that it will become a treatment for intractable epilepsy. Epilepsy refers to a group of chronic neurological disorders that are characterized by seizures, affecting over 50 million people,

neg: Clinical Ovarian Cancer & Other Gynecologic Malignancies Clinical Ovarian Cancer & Other Gynecologic Malignancies Clinical Ovarian Cancer & Other Gynecologic Malignancies was a peer-reviewed medical journal published by Elsevier. It covers research on the detection, diagnosis, prevention, and treatment of ovarian cancer. Specific areas of interest include clinical research and mechanistic approaches, drug sensitivity and resistance, gene and antisense therapy, pathology, markers, and prognostic indicators, chemoprevention strategies, multimodality therapy, and integration of various approaches. It has been replaced by Clinical Ovarian and other Gynecologic Cancer. The journal is abstracted and indexed in CINAHL, EMBASE, and Chemical Abstracts. The journal publishes editorials, original research papers, reviews, current treatment

neg: Molecular cloning an organ transplant. In this case, one or more specific tissues are targeted by direct treatment or by removal of the tissue, addition of the therapeutic gene or genes in the laboratory, and return of the treated cells to the patient. Clinical trials of somatic cell gene therapy began in the late 1990s, mostly for the treatment of cancers and blood, liver, and lung disorders. Despite a great deal of publicity and promises, the history of human gene therapy has been characterized by relatively limited success. The effect of introducing a gene into cells often promotes only partial and/or transient

neg: Gene therapy for epilepsy by the drug clozapine-N-oxide it suppressed seizures. The treatment had no detectable side effects and is, in principle, suited for clinical translation. Magnetofection is done through the use of super paramagnetic iron oxide nanoparticles coated with polyethylenimine. Iron oxide nanoparticles are ideal for biomedical applications in the body due to their biodegradable, cationic, non-toxic, and FDA-approved nature. Under gene transfer conditions, the receptors of interest are coated with the nanoparticles. The receptors will then home in and travel to the target of interest. Once the particle docks, the DNA is delivered to the cell via pinocytosis or endocytosis. Upon delivery,

neg: Genetic disorder ongoing battle with over 1800 gene therapy clinical trials having been completed, are ongoing, or have been approved worldwide. Despite this, most treatment options revolve around treating the symptoms of the disorders in an attempt to improve patient quality of life. Gene therapy refers to a form of treatment where a healthy gene is introduced to a patient. This should alleviate the defect caused by a faulty gene or slow the progression of disease. A major obstacle has been the delivery of genes to the appropriate cell, tissue, and organ affected by the disorder. How does one introduce a gene

neg: Melanoma in medicine do not use the word for benign tumors. Pharmacotherapy research for unresectable or metastatic malignant melanoma is ongoing. In clinical research setting other therapies, such as adoptive cell therapy or gene therapy, are being tested. Two kinds of experimental treatments developed at the National Cancer Institute (NCI), have been used in metastatic melanoma with tentative success. The first treatment involves adoptive cell therapy (ACT) using TILs immune cells (tumor infiltrating lymphocytes) isolated from a person's own melanoma tumor. These cells are grown in large numbers in a laboratory and returned to the patient after a treatment that temporarily

neg: Designer baby Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, on a four-year-old girl named, Ashanti DeSilva, she was treated for ADA-SCID. This is a disease that had left her defenseless against infections spreading throughout her body. Dr. W French Anderson was a major lead on this clinical trial, he worked for the National Heart, Lung, and Blood Institute Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy. The techniques

neg: Stefan Karlsson (professor) Cell Center, since 2003, and director of the Hemato-Linné Strategic Research Environment (2006-2016) funded through a 10-year Linneus grant from the Swedish Research Council. Dr. Karlsson also holds a consultant physician position at Skåne University Hospital, Sweden. Stefan Karlsson is recognized as an important contributor to the field of gene therapy. His early research focused on retroviral vector based gene correction of hematopoietic cells from monogenetic disorders, such as Gaucher’s disease and hemoglobinopathies. The results of these studies led to the first gene therapy clinical trial for the treatment of Gaucher’s disease (1995). He has also developed lentiviral vectors for

neg: Retinal gene therapy using lentiviral vectors Retinal gene therapy using lentiviral vectors Gene therapy using lentiviral vectors is being explored in early stage trials. In a Phase I clinical trial of three patients, two showed no improvement and one of them had some improvements. The study concluded further investigation is warranted for the use of the procedure to treat Leber's congenital amaurosis. Other early trials have been used to explore the treatments potential, including for therapeutic use of recombinant adeno-associated virus (rAAV) vectors. Many other possible viral vectors remain options for the treatment of various genetic disorders in the retina that lead to blindness. Retinal gene

neg: Stomach cancer early stomach cancer causes few symptoms, the disease is usually advanced when the diagnosis is made. Treatment for stomach cancer may include surgery, chemotherapy, and/or radiation therapy. New treatment approaches such as immunotherapy or gene therapy and improved ways of using current methods are being studied in clinical trials. Surgery remains the only curative therapy for stomach cancer. Of the different surgical techniques, endoscopic mucosal resection (EMR) is a treatment for early gastric cancer (tumor only involves the mucosa) that was pioneered in Japan and is available in the United States at some centers. In this procedure, the tumor, together

neg: Ornithine transcarbamylase deficiency deficiency is diagnosed using a combination of clinical findings and biochemical testing, while confirmation is often done using molecular genetics techniques. Once an individual has been diagnosed, the treatment goal is to avoid precipitating episodes that can cause an increased ammonia concentration. The most common treatment combines a low protein diet with nitrogen scavenging agents. Liver transplant is considered curative for this disease. Experimental trials of gene therapy using adenoviral vectors resulted in the death of one participant, Jesse Gelsinger, and have been discontinued. OTC deficiency can become apparent at any age. Early-onset OTC deficiency is most commonly found in

neg: Gene therapy into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew's blood. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed. Jesse Gelsinger's death in 1999 impeded gene therapy research in the US. As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices. The modified cancer

neg: Duchenne muscular dystrophy to develop therapies for DMD in the future. In 2007, researchers did the world's first clinical (viral-mediated) gene therapy trial for Duchenne MD. Biostrophin is a delivery vector for gene therapy in the treatment of Duchenne muscular dystrophy and Becker muscular dystrophy. Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy. The symptom of muscle weakness usually begins around the age of four in boys and worsens quickly. Typically muscle loss occurs first in the upper legs and pelvis followed by those of the upper arms. This can result in trouble standing up. Most are

neg: Gene therapy for epilepsy therapy continues to grow and show promising results for the treatment of epilepsy among other diseases, additional research needs to be done in ensuring patient safety, developing alternative methods for DNA delivery, and finding feasible methods for scaling up delivery volumes. Gene therapy for epilepsy Gene therapy is being studied for some forms of epilepsy. It relies on viral or non-viral vectors to deliver DNA or RNA to target brain areas where seizures arise, in order to prevent the development of epilepsy or to reduce the frequency and/or severity of seizures. Gene therapy has delivered promising results in early stage

neg: H19 (gene) suggestion of using the H19 promoter in gene therapy to drive the expression of cytotoxic genes in tumorigenic cells. Gene therapy trials utilizing the H19 promoter to drive the expression of cytotoxic genes are currently being tested on mice. A plasmid composed of the H19 gene regulatory sequences that drive the expression of the 'A' strand of Diphtheria Toxin (DT-A), is undergoing clinical testing as a treatment for superficial bladder cancer, ovarian cancer and pancreatic cancer. The plasmid, designated BC-819 (or DTA-H19), embodies a targeted therapy approach, in that the plasmid enters all dividing cells, but the DT-A expression is

neg: Fabry disease annual cost was about US$200,000 per patient, which is unaffordable to many patients around the world without enough insurance. Clinically the two products are generally perceived to be similar in effectiveness. Both are available in Europe and in many other parts of the world, but treatment costs remain very high. Besides these drugs, a gene therapy treatment is in clinical trials, with the technology licensed to AvroBio. Other treatments (oral chaperone therapy -Amicus-, plant-based ERT -Protalix-, substrate reduction therapy -Sanofi-Genzyme-, bio-better ERT -Codexis-, gene editing solution -Sangamo- are currently being researched. Pain associated with Fabry disease may be partially alleviated

neg: Clinical Genitourinary Cancer Clinical Genitourinary Cancer Clinical Genitourinary Cancer is a peer-reviewed medical journal published by Elsevier, and previously by CIG Media Group (Cancer Information Group). The journal publishes articles on detection, diagnosis, prevention, and treatment of genitourinary cancers. The main emphasis is on recent scientific developments in all areas related to genitourinary cancers. Specific areas of interest include clinical research and mechanistic approaches, drug sensitivity and resistance, gene and antisense therapy; pathology, markers, and prognostic indicators, chemoprevention strategies, multimodality therapy, and integration of various approaches. The journal was previously published as "Clinical Prostate Cancer" through September 2005. "Clinical Genitourinary Cancer" indexed in

neg: Gene therapy it was still largely an experimental technique. These include treatment of retinal diseases Leber's congenital amaurosis and choroideremia, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia (CLL), acute lymphocytic leukemia (ALL), multiple myeloma, haemophilia, and Parkinson's disease. Between 2013 and April 2014, US companies invested over $600 million in the field. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia. In 2012 Glybera, a treatment for a rare inherited disorder,

neg: X-linked severe combined immunodeficiency immune response (B-cell, T-cell, natural killer cell). Therefore, a BMT will result in a full immune reconstitution but there are aspects of BMT that need to be improved (i.e. GvHD). Gene therapy is another treatment option which is available only for clinical trials. X-linked SCID is a monogenic disorder, the IL2RG gene is mutated, so gene therapy will replace this mutated gene with a normal one. This will result in a normal functioning gamma chain protein of the interleukin receptor. In order to transfer a functional gene into the target cell, viral or non-viral vectors can be employed. Viral vectors,

neg: Clinical Ovarian Cancer & Other Gynecologic Malignancies reports, case reports, brief communications, current trials, translational medicine pieces, and a "Meeting Highlights" section. Clinical Ovarian Cancer & Other Gynecologic Malignancies Clinical Ovarian Cancer & Other Gynecologic Malignancies was a peer-reviewed medical journal published by Elsevier. It covers research on the detection, diagnosis, prevention, and treatment of ovarian cancer. Specific areas of interest include clinical research and mechanistic approaches, drug sensitivity and resistance, gene and antisense therapy, pathology, markers, and prognostic indicators, chemoprevention strategies, multimodality therapy, and integration of various approaches. It has been replaced by Clinical Ovarian and other Gynecologic Cancer. The journal is abstracted and indexed in

neg: Network of Excellence for Functional Biomaterials of a chronic model system using a functionalised biomaterials approach. Preclinical studies and preliminary data derived from clinical trials indicate the potential for the use of gene therapy or stem cell therapy for cardiovascular applications. At the NFB, the goal is to take gene or stem cell therapy and combine it with biomaterial delivery systems to enhance efficacy and improve control. The clinical targets are both ischemic muscle injuries, the first in the myocardium itself (myocardial infarction), and the second is in the lower limb (lower limb ischemia). The choice of gene in the case of biomaterial-mediated gene therapy is

neg: Gene doping entice me, saying things like, 'It'll help advance your research.' Some offered to pay me." He also told the "Times" that every time similar research is published he gets calls and that he explains that, even should the treatment became ready for use in people, which would take years, there would be serious risks, including death; he also said that even after he explains this, the athletes still want it. In 1999, the field of gene therapy was set back when Jesse Gelsinger died in a gene therapy clinical trial, suffering a massive inflammatory reaction to the drug. This led

neg: Leber's congenital amaurosis therapy to restore vision in LCA patients. In all three clinical trials, patients recovered functional vision without apparent side-effects. These studies, which used adeno-associated virus, have spawned a number of new studies investigating gene therapy for human retinal disease. The results of a phase 1 trial conducted by the University of Pennsylvania and Children’s Hospital of Philadelphia and published in 2009 showed sustained improvement in 12 subjects (ages 8 to 44) with RPE65-associated LCA after treatment with AAV2-hRPE65v2, a gene replacement therapy. Early intervention was associated with better results. In that study, patients were excluded based on the presence of

neg: Voretigene neparvovec approval of the treatment. The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received a pediatric disease priority review voucher. The first commercial sale of voretigene neparvovec -- the first for any gene therapy product in the US -- occurred in March 2018. The price of the treatment has been announced at $425,000 per eye. Voretigene neparvovec Voretigene neparvovec (Luxturna) is a novel gene therapy for the treatment of Leber's congenital amaurosis. It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first "in vivo" gene therapy approved by the

neg: Michael Jeffrey Aminoff implications for the treatment of these lesions. His later interest in movement disorders led him to show the utility of botulinum toxin in the treatment of various movement disorders, and then to study gene therapy in managing patients with Parkinson’s disease. Aminoff has devoted many years to medical education. As a director and chair of the American Board of Clinical Neurophysiology, of the American Board of Electrodiagnostic Medicine, and of the American Board of Psychiatry & Neurology, he was concerned with syllabus development, setting national guidelines and educational standards, and assessing competence in clinical neurophysiology and clinical neurology. He lectures

neg: Gene therapy for epilepsy by overexpressing somatostatin in specific cells, and increasing the GABAergic tone, it is possible to restore balance between inhibition and excitation. Kv1.1 is a voltage-gated potassium channel encoded by the KCNA1 gene. It is widely expressed in the brain and peripheral nerves, and plays a role in controlling the excitability of neurons and the amount of neurotransmitter released from axon terminals. Successful gene therapy using lentiviral delivery of KCNA1 has been reported in a rodent model of focal motor cortex epilepsy The treatment was well tolerated, with no detectable effect on sensorimotor coordination. A potential obstacle to clinical translation of

neg: Spark Therapeutics develop fidanacogene elaparvovec. Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients who carry non-functioning copies. In July, 2018 Pfizer said fidanacogene elaparvovec was entering late stage clinical trials. SPK-8011 is an experimental drug under investigation for treatment of hemophilia A. It is entering phase III clinical trials in the United States. The therapy transfers a working copy of the Factor VIII gene into patients who lack one. In Phase II clinical trials 2 of 7 patients receiving the highest dose of the drug

neg: Gene therapy and February 2016, over 2,300 clinical trials were conducted, with more than half of them in phase I. Not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients. Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect. Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. The first attempt, an unsuccessful one, at gene therapy (as well as the first

neg: Congenital dyserythropoietic anemia their own risks at this point in time. Bone marrow transplantation is the more used method between the two, whereas researchers are still trying to definitively establish the results of gene therapy treatment. It generally requires a 10/10 HLA matched donor, however, who is usually a sibling. As most patients do not have this, they must rely on gene therapy research to potentially provide them with an alternative. CDA at both clinical and genetic aspects are part of a heterogeneous group of genetic conditions. Gene therapy is still experimental and has largely only been tested in animal models until now.

neg: Gene therapy of the human retina for the treatment of choroideremia. Dr. Robert MacLaren of the University of Oxford, who lead the trial, co-developed the treatment with Dr. Miguel Seabra of the Imperial College, London. This Phase 1/2 trial used subretinal AAV to restore the REP gene in affected patients. Initial results of the trial were reported in January 2014 as promising as all six patients had better vision. Recent research has shown that AAV can successfully restore color vision to treat color blindness in adult monkeys. Although this treatment has not yet entered clinical trials for humans, this work was considered a breakthrough for the

neg: Ribozyme pyrophosphate (2). Fluorescence-activated_cell_sorting has also been used to engineering aptazymes. Ribozymes have been proposed and developed for the treatment of disease through gene therapy (3). One major challenge of using RNA based enzymes as a therapeutic is the short half-life of the catalytic RNA molecules in the body. To combat this, the 2’ position on the ribose is modified to improve RNA stability. One area of ribozyme gene therapy has been the inhibition of RNA-based viruses. A type of synthetic ribozyme directed against HIV RNA called gene shears has been developed and has entered clinical testing for HIV infection. Similarly,

neg: Critical limb ischemia example caused by an embolus whereas critical limb ischaemia is a late sign of a progressive chronic disease. Treatment mirrors that of other symptoms of peripheral artery disease, and includes modifying risk factors, revascularization via vascular bypass or angioplasty, and in the case of tissue loss, wound debridement. In 2011 pCMV-vegf165 was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia. In 2014, a landmark clinical trial was initiated to better understand the optimal revascularization technique for the treatment of CLI. As of April 2017, the Best Endovascular Versus Best Surgical

neg: Gene therapy immune response) removed using CRISPR-Cas9. A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy. In February Kite Pharma announced results from a clinical trial of

neg: Gene therapy for epilepsy treatment options to consider, and thus there is a strong need for the development of innovative methods for treating epilepsy. Through the use of viral vector gene transfer, with the purpose of delivering DNA or RNA to the epileptogenic zone, several neuropeptides, ion channels and neurotransmitter receptors have shown potential as transgenes for epilepsy treatment. Among vectors are adenovirus and adeno-associated virus vectors (AAV), which have the properties of high and efficient transduction, ease of production in high volumes, a wide range of hosts, and extended gene expression. Lentiviral vectors have also shown promise. Among challenges to clinical translation of

neg: Clinical neurochemistry time. Another treatment option is bromocriptine, which directly stimulates D Dopamine Receptors. Bromocriptine is less effective than L-Dopa in reducing symptoms, but provides less dyskinesia. Often, the two drugs are used in concert with one another. New approaches to treatment include deep brain simulation and cell transplantation with stem cells.Deep brain stimulation offsets symptoms rather than cures, and stem cell studies have been extremely disappointing, despite relative success with animal models. Gene therapy may provide a novel route to introduce new dopamine production via viral-medicated gene transfection, but clinical trials are yet to be completed. Huntington's Disease is a disease

neg: HIV/AIDS research cells (GM-HSPC). Though this study does involve several early stage clinical trials that have demonstrated the safety and feasibility of this technique only for HIV-1, none have resulted in improvement of the disease state itself. Therefore, this strategy is intended to go alongside already existing treatment techniques such as drugs and vaccines. However, future technology regarding this approach of single treatment cell therapy could potentially replace current therapy altogether as a functional or sterilizing cure to HIV-1. An additional study involves the use of genetically engineered CD34+ hematopoietic stem and progenitor cells. Experimental long-term in vivo HIV gene therapy have

neg: Gene therapy increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial. In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007. Leber's congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April. Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive

neg: Sandhoff disease Currently the government is testing several treatments including N-butyl-deoxynojirimycin in mice, as well as stem cell treatment in humans and other medical treatments recruiting test patients. A Sandhoff disease study showing proof of principle for gene therapy in a human model system using CRISPR and virus gene correction gives the chance for clinical trials to cure the disease. The ultra-rare occurrence is a main hurdle to overcome for clinical trials. Sandhoff disease is one of several forms of what was formerly known as amaurotic idiocy. This inherited disease is characterized by the accumulation of lipid-containing cells in the viscera and

neg: Gene therapy 24, 1993, and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena. In 1992 Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID). The success of a multi-center trial for

neg: Viral vector genes and lead to activation of oncogenes promoting the development of cancer, which raises concerns for possible applications of lentiviruses in gene therapy. However, studies have shown that lentivirus vectors have a lower tendency to integrate in places that potentially cause cancer than gamma-retroviral vectors. More specifically, one study found that lentiviral vectors did not cause either an increase in tumor incidence or an earlier onset of tumors in a mouse strain with a much higher incidence of tumors. Moreover, clinical trials that utilized lentiviral vectors to deliver gene therapy for the treatment of HIV experienced no increase in mutagenic

neg: Tay–Sachs disease (G444R) was found in the "HEXA" cDNA of the affected sheep. This mutation is a single nucleotide change at the end of exon 11, resulting in that exon's deletion (before translation) via splicing. The Tay–Sachs model provided by the Jacob sheep is the first to offer promise as a means for gene therapy clinical trials, which may prove useful for disease treatment in humans. Other experimental methods being researched involve substrate reduction therapy, which attempts to use alternative enzymes to increase the brain's catabolism of GM2 gangliosides to a point where residual degradative activity is sufficient to prevent substrate accumulation.

neg: Voretigene neparvovec Voretigene neparvovec Voretigene neparvovec (Luxturna) is a novel gene therapy for the treatment of Leber's congenital amaurosis. It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first "in vivo" gene therapy approved by the FDA. Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as an subretinal injection. Voretigene neparvovec is an AAV2 vector containing human RPE65 cDNA with

neg: Targeted molecular therapy for neuroblastoma inhibitors can also be used to treat another cause of neuroblastoma referred to as MYCN gene amplification. Amplification of the MYCN protein is a genetic mutation associated with neuroblastoma tumors. MYCN amplification is correlated with a specific mutation in ALK, referred to as the F1174L mutation. ALK inhibitors can target this mutation and suppress the MYCN protein in the tumor cell. The following is a list of ALK inhibitors currently in clinical trials for treatment of neuroblastoma: Crizotinib was the first of these drugs to enter clinical trials and is the sole available ALK inhibitor, approved by the FDA on

neg: Predictive marker in cancer therapy including better patient management minimizing unnecessary suffering from side effects with ultimately the wrong treatment choice, reducing loss of precious time whilst determining whether a therapy will provide any benefit, and a reduction in cost to both the patient and the wider health community. Predictive marker A predictive marker is a particular protein or gene that indicates sensitivity or resistance to a specific therapy. The use of predictive markers is becoming increasingly relevant in cancer therapy as it allows for better identification of patients who will respond positively to the therapy. In the clinical setting, predictive markers

neg: Neal S. Young Anfinsen (in the immunochemistry of hemoglobin) and Arthur Nienhuis (in globin gene regulation). He directed the first multicenter clinical trial using antithymocyte globulin (ATG)for the treatment of aplastic anemia in the United States. This therapy is now the standard treatment for the disease worldwide. He is credited with contributing to understanding the pathophysiology of the disease as immune-mediated and with developing immunotherapy for aplastic anemia that has dramatically improved survival rates for the disease. Prior to the introduction of immunotherapy in the early 1980s, the disease was largely fatal with survival rates around 10% one year following the diagnosis, whereas

neg: Friedreich's ataxia be about 40-50 years. Congestive heart failure and cardiac arrhythmia are the leading cause of death. However, some people with less severe features of FRDA live into their sixties or older. RG2833, a histone deacetylase inhibitor developed by Repligen, was acquired by BioMarin Pharmaceutical in January 2014. A phase Ib clinical trial with RG2833 has been successfully completed in 2014 and research continues. Research in gene therapy is another treatment under development. One study in mouse models showed that gene replacement therapy completely reversed the cardiac symptoms of Friedreich's Ataxia at the functional, cellular, and molecular levels. This provides a

neg: Batten disease pediatrician Frederick Batten, who first described it in 1903. Also known as Spielmeyer-Vogt-Sjögren-Batten disease, it is the most common form of a group of disorders called neuronal ceroid lipofuscinosis (NCL). Although Batten disease is usually regarded as the juvenile form of NCL, some physicians use the term Batten disease to describe all forms of NCL. In June 1987, a phase-I clinical trial was launched at Weill Cornell Medical College of Cornell University to study a gene therapy method for treatment of the signs and symptoms of LINCL. The experimental drug works by delivering a gene transfer vector called AAV2CUhCLN2 to

neg: Gene therapy became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission. Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered – replacing or disrupting defective genes. Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase. DNA must be administered, reach the damaged cells, enter the cell and

neg: Gene therapy that gene will not be produced. Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus. In 2003, it was approved in China for the treatment of head and neck squamous cell carcinoma. In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system. In May a team reported a way to prevent the immune system from rejecting

neg: Choroideremia modification of Rab proteins. While the complete mechanism of disease is not fully understood, the lack of a functional protein in the retina results in cell death and the gradual deterioration of the retinal pigment epithelium (RPE), photoreceptors, and the choroid. As of 2018, there is no treatment for choroideremia; however, retinal gene therapy clinical trials have demonstrated a possible treatment. Since the "CHM" gene is located on the X chromosome, symptoms are seen almost exclusively in men. While there are a few exceptions, female carriers have a noticeable lack of pigmentation in the RPE but do not experience any

neg: European Society of Gene and Cell Therapy European Society of Gene and Cell Therapy The European Society of Gene and Cell Therapy (ESGCT) is a non-profit organisation for educational and scientific purposes. The aim of ESGCT is to promote fundamental and clinical research in gene therapy, cell therapy, and genetic vaccines by facilitating education, the exchange of information and technology and by serving as a professional adviser to stakeholder communities and regulatory bodies in Europe. ESGCT was founded in January 1992 as the European Working Group on Human Gene Transfer and Therapy (EWGT). The Society was renamed the European Society of Gene Therapy (ESGT) in 1998. In

neg: Therapy primary therapy, or front-line therapy) is the first therapy that will be tried. Its priority over other options is usually either: (1) formally recommended on the basis of clinical trial evidence for its best-available combination of efficacy, safety, and tolerability or (2) chosen based on the clinical experience of the physician. If a first-line therapy either fails to resolve the issue or produces intolerable side effects, additional (second-line) therapies may be substituted or added to the treatment regimen, followed by third-line therapies, and so on. An example of a context in which the formalization of treatment algorithms and the ranking

neg: European Society of Gene and Cell Therapy Annual Congress of the ESGCT take place in a different location in Europe every year to reflect the international character of the society. Since the first congress in 1993, Congresses have taken place in European Society of Gene and Cell Therapy The European Society of Gene and Cell Therapy (ESGCT) is a non-profit organisation for educational and scientific purposes. The aim of ESGCT is to promote fundamental and clinical research in gene therapy, cell therapy, and genetic vaccines by facilitating education, the exchange of information and technology and by serving as a professional adviser to stakeholder communities and regulatory bodies

neg: Gene therapy in Parkinson's disease of dopamine or protect the nervous system dopamine neurons from degeneration. There are some important reasons for focusing on gene therapy as a treatment for PD. First of all, currently there is no cure for this disease. Secondly, some genes have been identified which can modulate the neuron phenotype or act as neuroprotective agents. Also, the environment of the brain cannot afford repeated injections into the region where the substantia nigra meets the striatum, the nigrostriatum. Therefore, gene therapy could be a single treatment appealing, viral vectors used in the therapy are diffusible and capable to do transduction of the

neg: NUT midline carcinoma Hassall corpuscle of the thymus. The defining feature of NMCs is rearrangement of the "NUT" gene. Most common is a translocation involving the BRD4 gene and NUT gene (t(15;19)(q13;p13.1)). NUT midline carcinoma is very resistant to standard chemotherapy treatments. The tumor may initially respond to therapy, and then rapid recurrence is experienced. A multimodality approach to treatment is advocated, especially since most patients present with advanced disease. Treatment must be tailored to the individual patient, with several promising new targeted molecular therapies in clinical trials. Specific molecular targeted therapies (BET inhibitors and histone deacetylase inhibitors (HDACi)) may help to yield

neg: Gene therapy cells. In November researchers reported on the use of VRX496, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial, five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. A single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or

neg: Sleep paralysis was done using a culturally sensitive cognitive-behavior therapy called CA-CBT. The work focuses on psycho-education and modifying catastrophic cognitions about the sleep paralysis attack. This approach has previously been used to treat sleep paralysis in Egypt, although clinical trials are lacking. The first published psychosocial treatment for recurrent isolated sleep paralysis was cognitive-behavior therapy for isolated sleep paralysis (CBT-ISP). CBT-ISP is manualized, has an adherence manual for research purposes, and is intended to both prevent and disrupt ISP episodes. It begins with self-monitoring of symptoms, cognitive restructuring of maladaptive thoughts relevant to ISP (e.g., "the paralysis will be permanent"), and

neg: Mark Batshaw treatment, involving sodium phenylbutyrate and glycerol phenylbutyrate, is still in use. In 1988, Batshaw moved from Hopkins to the University of Pennsylvania where he began a collaboration with James Wilson. They developed an adenovirus vector and started a gene therapy clinical trial that resulted in the death of a patient, Jesse Gelsinger, in 1999 and a subsequent slowdown of the development of gene therapy. In the past decade, the field has recovered and Wilson and Batshaw’s teams have developed an adeno-associated virus vector that has been successful in preclinical studies of an animal model of urea cycle disorders. Mark Batshaw

neg: Gene doping Gene doping Gene doping is the hypothetical non-therapeutic use of gene therapy by athletes in order to improve their performance in those sporting events which prohibit such applications of genetic modification technology, and for reasons other than the treatment of disease. , there is no evidence that gene doping has been used for athletic performance-enhancement in any sporting events. Gene doping would involve the use of gene transfer to increase or decrease gene expression and protein biosynthesis of a specific human protein; this could be done by directly injecting the gene carrier into the person, or by taking cells from

neg: The Park Centre for Mental Health patients and treatment with drugs. He noted:"Modern treatment demands exhaustive mental and clinical case histories, as well as completely thorough physical examination. This cannot be done by a skeleton staff, however willing".This approach also brought degrees of specialisation among the staff and hospital procedures. Stafford advocated the separation of chronic wards from those dealing with admission, convalescence and hospital cases. He believed that mental illness "demands active therapy, and treatment must not become merely custodial" and urged the use of new types of treatment such as insulin, cardiazol and electrotherapy. The first building at Wolston Park to reflect Stafford's modern

neg: Center for Cell and Gene Therapy Phase I/II trials, while the GMP Clinical Research Lab prepares patient components for clinical trials. The Gene Vector Lab was one of only three National Gene Vector Laboratories until that entity was replaced by the National Gene Vector Biorepository in 2008. The Research Lab is a member of the Production Assistance for Cell Therapies (PACT). The Stem Cell Transplantation Program has two units. The pediatric unit has more than 16,000 square feet on the eighth floor of Texas Children’s Hospital’s West Tower. The 30,000-square foot adult unit is in The Methodist Hospital’s Main Tower. Center for Cell and Gene Therapy

neg: Antisense therapy membranes is also difficult. Therefore, most clinical candidates have modified DNA "backbones", or the nucleobase or sugar moieties of the nucleotides are altered. Additionally, other molecules may be conjugated to antisense molecules in order to improve their ability to target certain cells or to cross barriers like cell membranes or the blood brain barrier. Antisense therapy Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical

neg: Mentalization-based treatment on mentalizing in therapy, appear to facilitate change by leaving people more open to learning outside of therapy, in the social interactions of their day-to-day lives. Fonagy, Bateman, and colleagues have done extensive outcome research on MBT for borderline personality disorder. The first randomized, controlled trial was published in 1999, concerning MBT delivered in a partial hospital setting. The results showed real-world clinical effectiveness that compared favorably with existing treatments for BPD. A follow-up study published in 2003 demonstrated that MBT is cost-effective. Encouraging results were also found in an 18-month study, in which subjects were randomly assigned to an

neg: Center for Cell and Gene Therapy Gene Therapy conducts research into numerous diseases, including but not limited to pediatric cancers, diabetes, HIV, glioma and cardiovascular disease. The Center has laboratory space in both Baylor College of Medicine and Texas Children’s Hospital, and clinical units in Texas Children’s and Methodist Hospitals. The Texas Children’s Hospital is home to the Center’s Translational Research Labs and Good Manufacturing Practice (GMP) Laboratories. The Center for Cell and Gene Therapy has the largest academic GMP facility in the world, with 8,600 square feet of Class 10,000 (ISO7) cleanroom space. The GMP Gene Vector Lab produces clinical grade vectors for use in

neg: Gene doping human enhancement at several sessions. In 2003, the field of gene therapy took a step forward and a step back; first gene therapy drug was approved, Gendicine, which was approved in China for the treatment of certain cancers, but children in France who had seemingly been effective treated with gene therapy for severe combined immunodeficiency (non-human) began developing leukemia. In 2003 the BALCO scandal became public, in which chemists, trainers and athletes conspired to evade doping controls with new and undetectable doping substances. In 2003 the World Doping Agency proactively added gene doping to the list of banned doping practices.

neg: Gene therapy results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects. In September researchers were able to give trichromatic vision to squirrel monkeys. In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder. An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two

neg: Cognitive Retention Therapy is currently undergoing formal clinical studies, but has been presented at different seminars and conventions including the 28th National Conference of the Alzheimer Society of Canada in Toronto, Ontario; and has received positive feedback as a plausible treatment option. Cognitive Retention Therapy Cognitive Retention Therapy (CRT) also known as the Ashby Memory Method is a Cognitive therapy for dementia, based on the research of Dr. Mira Ashby. It is adapted from her programs for brain injury rehabilitation, for which she won the Order of Canada in 1984. CRT is a specifically tailored program to work for the damage done by

neg: Clinical Colorectal Cancer Clinical Colorectal Cancer Clinical Colorectal Cancer is a peer-reviewed medical journal published by CIG Media Group (Cancer Information Group) from 2001 to 2010 and by Elsevier since 2011. It publishes original articles describing various aspects of clinical and translational research of gastrointestinal cancers. The journal is devoted to articles on detection, diagnosis, prevention, and treatment of colorectal, pancreatic, liver, and other gastrointestinal cancers. The main emphasis is on recent scientific developments in all areas related to gastrointestinal cancers. Specific areas of interest include clinical research and mechanistic approaches; drug sensitivity and resistance; gene and antisense therapy; pathology, markers, and prognostic

neg: Claudio Bordignon Claudio Bordignon In 1992 Doctor Claudio Bordignon working at the Vita-Salute San Raffaele University, Milan, Italy performed the first procedure of gene therapy using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. This was a world first, but was unfortunately unsuccessful because it did not lead to sustained correction of the hematopoietic stem cells. In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (SCID). He expanded this work to stem cell gene therapy of other genetic diseases and AIDS, and for the immunotherapy of cancer.

neg: Stretta procedure treatment based on the 2012 review. In 2015 three reviews were published discussing the relative safety and effectiveness of the procedure compared with other endoscopic procedures delivered through the mouth, drug treatment (generally proton-pump inhibitors), and fundoplication. One was a systematic review and meta-analysis of clinical trials conducted with Stretta, done in response to the 2013 SAGES review (which did not include meta-analysis). This review found that quality of studies that had been conducted was generally poor, and that compared with sham therapy (used a placebo for medical device clinical trials), the procedure did not change time spent at a

neg: Gene therapy the treatment of blindness due to Leber's congenital amaurosis. The price of this treatment was 850,000 US dollars for both eyes. Speculated uses for gene therapy include: Athletes might adopt gene therapy technologies to improve their performance. Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports. Genetic engineering could be used to cure diseases, but also to change physical

neg: Niemann–Pick disease, type C in January 2012. Due to unprecedented collaboration between individual physicians and parents of children afflicted with NPC, approximately 15 patients worldwide have received HPbCD cyclodextrin therapy under compassionate use treatment protocols. Treatment involves a combination of intravenous therapy (IV), intrathecal therapy (IT) and intracerebroventricular (ICV) cyclodextrin therapy. On January 23, 2013, a formal clinical trial to evaluate HPβCD cyclodextrin therapy as a treatment for Niemann–Pick disease, type C was announced by scientists from the NIH's National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). A Phase I clinical

neg: Moss bioreactor is part of the human complement system. Defects in the corresponding gene are associated with human diseases such as severe kidney and retinal disorders. Biologically active recombinant factor H was produced in a moss bioreactor for the first time in 2011. The enzyme alpha-galactosidase now is allowed to be produced in moss bioreactors by the German Federal Institute for Drugs and Medical Devices. It will be tested as enzyme replacement therapy in the treatment of Fabry's disease. The clinical trial phase 1 was completed in 2017. Moss bioreactor A moss bioreactor is a photobioreactor used for the cultivation and propagation

neg: Clinical significance significance). It is very possible to have a treatment that yields a significant difference and medium or large effect sizes, but does not move a patient from dysfunctional to functional. Within psychology and psychotherapy, clinical significance was first proposed by Jacobson, Follette, and Revenstorf as a way to answer the question, is a therapy or treatment effective enough such that a client does not meet the criteria for a diagnosis? Jacobson and Truax later defined clinical significance as “the extent to which therapy moves someone outside the range of the dysfunctional population or within the range of the functional population.”

neg: Ornithine transcarbamylase deficiency as preventative treatment with nitrogen scavenging agents such as sodium benzoate. The goal is to minimize the nitrogen intake while allowing waste nitrogen to be excreted by alternate pathways. Arginine is typically supplemented as well, in an effort to improve the overall function of the urea cycle. If a hyperammonemic episode occurs, the aim of treatment is to reduce the individual's ammonia levels as soon as possible. In extreme cases, this can involve hemodialysis. Gene therapy had been considered a possibility for curative treatment for OTC deficiency, and clinical trials were taking place at the University of Pennsylvania in the

neg: University of Arkansas for Medical Sciences the Arkansas Biosciences Institute and the UAMS Bioventures Business Incubator. UAMS is ranked in the top 20% of all US Colleges & Universities in research funding from Federal Government. In 2016, the university’s research funding was more than $111.9 million. Clinical researchers at UAMS performed the first outpatient bone marrow transplant in the US, as well as the first gene therapy for multiple myeloma in the US. They are also home to a number of robotic, experimental, and advanced treatment facilities found nowhere else in the state of Arkansas. The school has the second highest yield of all United States

neg: Gene therapy 10 participants in July. The study was expected to continue until 2015. In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis. The recommendation was endorsed by the European Commission in November 2012 and commercial rollout began in late 2014. Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012, revised to $1 million in 2015, making it the most expensive medicine in the world

neg: Gene therapy of the human retina reported results of clinical trials using adeno-associated virus for LCA. In these studies, an AAV vector encoding the RPE65 gene was delivered via a "subretinal injection", where a small amount of fluid is injected underneath the retina in a short surgical procedure. Development continued, and in December 2017 the FDA approved Voretigene neparvovec (Luxturna), an adeno-associated virus vector-based gene therapy for children and adults with biallelic RPE65 gene mutations responsible for retinal dystrophy, including Leber congenital amaurosis. People must have viable retinal cells as a prerequisite for the intraocular administration of the drug. Following the successful clinical trials in LCA,

neg: Breast-conserving surgery lymph nodes is sufficient to recommend treatment. In the selection of patients for breast conservation treatment with radiation, there are some absolute and relative contraindications. Absolute contraindications, which are reasons why the procedure absolutely cannot be done, include: Breast conserving therapy (BCT) is the process of following up BCS with moderate dose radiation therapy in order to eliminate residual disease in the breast and decrease the likelihood of disease recurrence. The goal of this process is to give the patient an equivalent survival rate to mastectomy while limiting cosmetic damage caused by surgery. Six recent clinical trials concluded that BCT

neg: Claudio Bordignon a Delivery System for Antigens 11-20-2008 Claudio Bordignon In 1992 Doctor Claudio Bordignon working at the Vita-Salute San Raffaele University, Milan, Italy performed the first procedure of gene therapy using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. This was a world first, but was unfortunately unsuccessful because it did not lead to sustained correction of the hematopoietic stem cells. In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (SCID). He expanded this work to stem cell gene therapy of other genetic diseases and AIDS,

neg: Lipoprotein lipase deficiency severe or multiple pancreatitis attacks despite dietary fat restrictions. It was the first gene therapy to receive marketing authorization in Europe; it was priced at about $1 million per treatment, and as of 2016, only one person had been treated with it commercially. A total of 31 patients were treated with Glybera, most for free in clinical trials before the drug was taken off the market. The disorder affects about 1 out of 1,000,000 people; however, epidemiological data are limited and there are regional differences due to cofounder effect (e.g. in Canada) or intermarriage. Lipoprotein lipase deficiency Lipoprotein lipase deficiency

neg: Robert J. Desnick Robert J. Desnick Robert J. Desnick, Ph.D., M.D., (born July 12, 1943) is a human geneticist whose basic and translational research accomplishments include significant discoveries in genomics, pharmacogenetics, gene therapy, personalized medicine, and the treatment of genetic diseases. His translational research has led to the development of enzyme replacement therapy for Fabry disease and Niemann-Pick disease Type B (in “breakthrough” clinical trials). He was the co-founder of Amicus Therapeutics, a biopharmaceutical company developing pharmacologic chaperone therapies (Galafold approved 2018), and served as the Chairman of the Scientific Advisory Committee (SAC) of Synageva BioPharma (2015 takeover by Alexion for $8.4 billion)

neg: Chronic lymphocytic leukemia remission, while the presence of leukemia in the third patient reduced by 70%. One of the patients had been diagnosed with CLL for 13 years, and his treatment was failing before he participated in the clinical trial. One week after the T cells were injected, the leukemia cells in his blood had disappeared. The T cells were still found in the bloodstream of the patients six months after the procedure, meaning they would be able to fight the disease should leukemia cells return. This was the first time scientists "have used gene therapy to successfully destroy cancer tumors in patients