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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Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden's rare, inherited immune deficiency. But it's not available to him.
Jun 21, 2024
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Researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.
Jun 15, 2024
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A viral gene therapy developed by University at Buffalo researchers has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...
Jun 13, 2024
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Researchers at Columbia University College of Dental Medicine have developed an exosome-based platform, "safeEXO-Cas," that significantly enhances the delivery of CRISPR/Cas9 genome editing components to specific cells and ...
Jun 7, 2024
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A novel gene therapy designed to target a form of inherited deafness has restored hearing function in five children who were treated in both ears. The children also experienced better speech perception and gained the ability ...
Jun 5, 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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