Some cases where gene therapy has been successful are in the breakthrough with the disease Amytrophic Lateral Sclerosis. |
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This gave rise to the genetic revolution, including recombinant DNA technology and gene therapy. |
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If this research is going to succeed, if we're talking about gene therapy, you have to clone some cells, don't you? |
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The grant will fund two clinical trials, three preclinical research studies, and a training program in gene therapy. |
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An international team of scientists has managed to cure a child with severe combined immunodeficiency using gene therapy. |
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One of the objectives was to assess the safety of administering the gene therapy locally to the pancreatic tumour. |
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One important application is gene therapy, where it is necessary to deliver a gene or a synthetic oligonucleotide into the cell. |
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No treatments for these disorders now exist although gene therapy may be a future possibility. |
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New cell selection strategies look likely to improve the success of gene therapy for blood disorders. |
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It makes sense to use gene therapy to program stem cells to produce the exact growth factors needed for each purpose. |
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The biggest benefits for health and longevity will come from cell therapy and gene therapy. |
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In laboratory studies, this type of gene therapy has proved almost completely effective. |
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Scientists hope that these unlucky fish will help them develop drug treatments and gene therapy for leukaemia. |
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The major problems with gene therapy relate to the delivery of new genetic instructions to the appropriate body targets. |
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At present, gene therapy is being contemplated for both genetic and acquired diseases. |
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As lung damage is the major cause of illness and death in cystic fibrosis, initially, gene therapy is focusing on delivering genes to the lungs. |
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Many people believe that gene therapy and genetic screening of embryos is dangerous. |
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In the near future gene therapy may allow genes important in healing to be delivered directly into a wound. |
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In the US, gene therapy is currently only being tested for the treatment of diseases that have no other cures. |
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Another possibility would be to do gene therapy to stem cells that are capable of becoming liver cells. |
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A US donor was finally located at the same time that gene therapy became available. |
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The most logical approach to gene therapy is the correction of a single gene defect, which causes the disease phenotype. |
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In the long run, I fully expect gene therapy and antisense to fix what can be fixed at the genomic level. |
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Is gene therapy, the medical use of genes to repair an illness or injury, acceptable for Olympic athletes? |
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The teams are only the third in the world to claim success in curing anyone using gene therapy. |
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In gene therapy, orthopedic surgeons harvest cells from a patient, modify them and return them to a particular area of the body. |
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One reason is that cell therapy and gene therapy development are both still in their infancy. |
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If we try to give gene therapy intravenously, it probably won't make it to all the tumour cells. |
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More than any other area of genetics, then, the beneficial possibilities of gene therapy have been much vaunted. |
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But the future is looking brighter after Holly was tested in New York for her suitability to trial a radical gene therapy for the disease. |
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As a vehicle for gene therapy, the retrovirus is modified so that it cannot produce infectious virus after entering the cell. |
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Combined with a beta cell growth factor called Btc, the gene therapy complete cured the mice's diabetes for at least four months. |
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The researchers at first tried to deliver the gene therapy while the patients were sedated but awake. |
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The culprit may be the gene therapy company, perceived impersonally as located far away and likely to be backstopped by an insurance policy. |
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They have created a national commission that centralizes all information relating to gene therapy. |
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This work represents a vital step in the development of a gene therapy for this genetic disease which remains at present untreatable. |
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Already cloning embryos, using aborted foetuses, gene swapping and gene therapy will mean the long term impacts will be immense on our everyday living. |
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I was surprised to hear of Brando's strange obsession with living forever, even studying gene therapy and cryogenics in an effort to prolong his life. |
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Two children affected with a lethal brain disorder, adrenoleukodystrophy, have been successfully treated with a new gene therapy vector. |
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Success of gene therapy for adrenoleukodystrophy: a new victory thanks to you! |
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A pioneering form of gene therapy has cured deafness in guinea pigs. |
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Moreover, gene therapy seemed like the only possible therapy for many genetic diseases. |
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These data position gene therapy as an attractive treatment option for inhibitors-positive haemophilic patients. |
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A gene therapy approach was also tested: the trans-splicing of the last exon of titin. |
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Therapies geared to reducing oxidative stress are in development, including gene therapy and new pharmacotherapy. |
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Somatic gene therapy aims to correct the genetic defects in the somatic cells and to produce an effect restricted to the person treated. |
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Baculovirus was initially developed as technology to be used in gene therapy, due to its non-mammalian non-cytological properties. |
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It would be ironic if the first successful application of gene therapy were to people who are among the fittest on the planet. |
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None will be trickier than the expected emergence of gene therapy, starting with treatments for, say, muscular dystrophy. |
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No gene therapy trials may be carried out which result in modifications to the subject's germ line genetic identity. |
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With the exception of gene therapy, physical risks in genetic research are generally similar to those seen in other forms of research. |
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Paradoxically, at the same time, scientific magazines were reporting on very promising results in gene therapy obtained by a French group. |
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The first clinical gene therapy is underway to correct an enzyme deficiency called ADA in children. |
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This injectable gene therapy could help delay amputations in a statistically significant manner. |
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Recombinant retroviruses are a widely used gene therapy vector because of their ability to permanently integrate a therapeutic transgene into the genome of a target cell. |
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Tissue engineering, gene therapy, therapeutic proteins, microelectromechanical systems, robotics, and bioinformatics are creating a myriad of disruptive technologies. |
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Gene vaccines borrow from both traditional vaccinology and gene therapy. |
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Host-pathogen interactions are studied in the light of the immune response, of various structure-function studies, of development of vaccines and viral vectors for gene therapy and food salubrity. |
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This non-invasive strategy for gene transfer into the CNS represents a significant advance in treatment of diseases of the motoneuron by gene therapy. |
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More fundamental research is also being deployed to face challenges such as overcoming the blood-brain barrier or achieving effective gene therapy. |
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The grant is repayable once revenues are generated from sales of ex vivo IL-10 gene therapy. |
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The idea behind exon skipping is an ingenious one: a disease can be cured, or at least ameliorated, not by replacing a defective gene, as is done in gene therapy, but by correcting it. |
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If it is moral, as it surely would be, to remedy his condition by somatic cell gene therapy, why not cure it once and for all by germline techniques? |
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But biologic treatments such as angiogenesis inhibition, viral oncolysis, and gene therapy are more promising in the long term. |
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Within such restrictions, the possible use of gene therapy involving the somatic cells of a sick patient is not basically different from organ transplants, bone marrow in particular. |
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Moreover it is impossible to exclude the risk of affecting germinal cells, and thus effectively performing germinal gene therapy, when using these viral vectors, particularly in the case of an in utero embryo. |
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In the meantime, we have now refocused our efforts on the area of conventional gene therapy using viral vector delivery of a normal canine FVIII gene to hemophilic mice and dogs. |
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His laboratory also collaborates on gene therapy projects and conducts its own research such as gene therapy with AAV in Leber's congenital amaurosis, a rare disease of the retina. |
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This trial will take place in London under the direction of Professor Adrian Thrasher, and will use a gene therapy protocol ex vivo with a lentivirus vector. |
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For example, gene therapy might be used to boost proinsulin expression in bone marrow cells in a laboratory, which could then be reinfused into people at risk for type 1 diabetes. |
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One example of a targeted delivery system is a virus that is used as a vector to transfect a foreign gene into a cell for the purpose of immunization or for gene therapy. |
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Somatic cell gene therapy systems work only so long as the transduced cells either live or give rise to new cells in which the inserted gene is also present. |
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And scientists successfully have used gene therapy to treat the fatal brain disorder adrenoleukodystrophy in children. |
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But the evidence suggests that EPO produced as a result of gene therapy will be far harder to distinguish. In fact, EPO doping may already have happened. |
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Use of a NK-1 receptor antagonist in the preparation of a medicament for preventing or treating the neurogenic inflammation caused or potentiated by the administration of viral vectors in gene therapy in a mammal. |
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Community legislation on pharmaceutical products already applies to gene therapy and to the medicines used in cell therapy in terms of general principles. |
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Nanoparticle carriers developed could potentially carry small molecules, gene therapy agents, antibodies as well as contrasting agents used in tumor imaging. |
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Anti-thrombin peptides deliverable through gene therapy. |
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Refined human artificial chromosome vectors for gene therapy and animal transgenesis. |
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Already, the US Food and Drug Administration has approved the first gene therapy trial, albeit for a rare genetic malady known as adenosine deaminase deficiency, an immunodeficiency disease. |
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This enumeration of actions will also make it possible to cover medicinal products such as gene therapy, radiopharmaceutical products as well as certain medicinal products for topical use. |
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Gelsinger participated in a clinical trial designed to test a potential gene therapy for ornithine transcarbamylase deficiency. |
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At the heart of bluebird bio's product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. |
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Yet gene therapy also offers immense promise. |
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Other gene therapy researchers expressed cautious optimism. |
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In gene therapy, this process is hijacked. |
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Researchers and gene therapy companies became skittish. |
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Thirteen per cent of boys with DMD could be treated with this specific 'antisense' gene therapy, the largest group by a single antisense. |
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As the largely disappointing experience with gene therapy should remind us, many highly vaunted, scientific techniques frequently failed to yield the promised results. |
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In addition to her work at CRDP, Geneviève Cardinal is completing a master's degree on a subject that we're hearing a lot about recently: gene therapy. |
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Our Report could, of course, maintain the focus on genetic disease as traditionally understood by simply defining human gene therapy as therapy directed at such problems. |
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Dr. Foldvari's approach involves producing microscopic delivery molecules specifically designed for gene therapy that are safer for patients and easier to mass produce than viral delivery systems. |
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However, some reports had suggested that RNAi gene therapy might induce an immune reaction or switch off the wrong gene or genes. |
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Discover how disease can be treated with gene therapy, the introduction of a new gene into a cell that changes the expression of a person's genes to cure or prevent genetic diseases. |
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While they may be potentially very useful in vaccine and gene therapy, they can also serve as delivery vehicles for dangerous toxins or bioregulators. |
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In addition, the Italian delegation wishes to make it clear that germ gene therapy cannot be recognized as patentable and legitimate, under the 13th recital of the common position. |
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Like the human genome project, and the gene therapy that has developed from its decoding, this research will make it possible to design extremely high performance materials and to prolong their useful life. |
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While those who dream of the day when gene therapy might reduce the frequency of treatments to once a year or less may find this unimpressive, Peter Larson says that the benefits would be critical. |
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Presently, gene therapy and nutraceutical supplementation are popular areas of ongoing research. |
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Adenoviruses are genetically modified viruses used in gene therapy to treat cystic fibrosis, cancer and other diseases. |
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The approach uses gene therapy to regrow cilia, cell structures that are essential for olfactory function. |
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One potential solution is to combine radiation treatment with sustained gene therapy to disable a number of the common receptors used by the virus, but there is still some way to go to prove this is safe. |
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Human pharmacodynamic studies shall address the expression and function of the nucleic acid sequence following administration of the gene therapy medicinal product. |
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This may be accomplished by beta cell gene therapy or by drugs that interfere with this pathway in order to maintain normal beta cell function. |
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The progress of molecular biology and a better understanding of the physiopathology of myopathies has now made some gene therapy options credible. |
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The term human gene therapy came into use because it identified as presumptively beneficent, through the use of the word therapy, technologies that might have provoked more opposition if called human genetic engineering. |
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The conclusion that gene therapy will be most frequently used for treatments other than conventional monogenic disease leaps out from the present evidence and trends. |
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This virus, which integrates naturally into the host cell genome, is particularly interesting for gene therapy because of its capacity to transport the transgene into both quiescent and proliferating cells. |
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Bartus and his associates at Ceregene developed a viral gene therapy vector bioengineered to express only the gene for human nerve growth factor. |
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Many pre-clinical studies of hemophilia A gene therapy have now been reported, most of which have utilized viral vector-mediated transgene delivery approaches in hemophilic mice or dogs. |
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There are growing numbers of hemophilic mice and dogs that have had their bleeding problems successfully treated with gene therapy for periods of several years. |
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Oncology, infectious diseases, genetic disorders, cardiovascular diseases and ophthalmological indications are the most active gene therapy pipeline sectors. |
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Researchers are now developing gene therapy that would boost the production of LEM protein, increasing the number of T-cells to attack cancer cells. |
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The gene therapy, called ProSavin, restored the monkeys' levels of dopamine, corrected motor problems and prevented dyskinesias, as jerkiness, rigidity and tremor are called. |
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Human adenoviruses are excellent tools for efficient gene transfer into a variety of cells and are currently widely used as vectors for gene therapy. |
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The development of DNA vaccines and gene therapy vectors as well as genetic vaccination for monoclonal antibodies and antisera also profit from GENEART's technology. |
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All current uses of gene therapy involve somatic cell gene transfers. |
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A recent article in the scholarly journal Nature Medicine announced auditory hair cell replacement and hearing improvement after using gene therapy in deaf lab animals. |
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Although the initial studies of gene therapy were conducted mostly on monogenetic diseases, the focus of the scientists rapidly shifted towards cancer. |
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As such, it delivers diverse protocols that cover topics ranging from the basic pathophysiology of cystic fibrosis to animal models and gene therapy. |
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Although urologists were underusing new minimally invasive treatments, they were aware of emerging treatment options, including gene therapy and autoimmune therapy. |
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