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Gene therapy is a medical technology ... the first gene therapy widely accepted as a success was demonstrated in a ... for example in a tumor suppressor gene, ...
About 6.7 million adults in the U.S. have heart failure, statistics show. "Gene therapy has emerged as a new modality that could interfere or modify the expression of several proteins, thus it ...
An experimental gene therapy appears to have been successful in halting the progression of one woman's advanced pancreatic cancer, doctors reported Wednesday. ... The apparent success of the ...
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV).
The Bespoke Gene Therapy Consortium, was organized by the Foundation for the National Institutes of Health and includes the FDA, various NIH institutes, and several drug companies and nonprofits.
The safety of gene therapy treatment is of utmost concern, especially during clinical trials when off-target modifications can block the further development of a candidate product. [55] Perhaps the most well-known example of modern gene therapy is CAR-T therapy, which is used for the treatment of B-cell lymphoma.
Scientists have characterized the role of thousands of mutations in the BRCA2 cancer gene, findings that may help reassure worried patients about their cancer risk or guide doctors toward better ...
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