The protein tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells' internal structures. These tangles have ...
Jul 23, 2024
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A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those ...
Jul 17, 2024
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In biomedical research involving human subjects, research ethics committees around the world have traditionally emphasized individual rights and protections for participants, including informed consent. The emerging field ...
Jul 12, 2024
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Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can trigger innate immune pathways, leading to the initiation of adaptive immune responses. A new review article published in the journal Human Gene ...
Jul 10, 2024
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A review article provides a comprehensive overview of the biological mechanisms of suppressor tRNA, traces the development history of related research, explains the advantages and disadvantages of suppressor tRNA therapy ...
Jul 8, 2024
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When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids) in March 2022, the course of his condition was dramatically altered.
Jun 28, 2024
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Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades. But decades can feel ...
Jun 27, 2024
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A new method developed by a research team led by Rice University's Jerzy Szablowski offers hope for treating brain disorders through gene therapy. The innovative approach could transform treatments for inherited conditions ...
Jun 26, 2024
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In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or ...
Jun 25, 2024
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The U.S. Food and Drug Administration has expanded the approval of Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in individuals ≥4 ...
Jun 25, 2024
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Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
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