Gene Therapy Approaches for the Treatment of Hemophilia B.
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ABSTRACT:
SUBMITTER: Soroka AB
PROVIDER: S-EPMC10341900 | biostudies-literature | 2023 Jun
REPOSITORIES: biostudies-literature
Publications
Gene Therapy Approaches for the Treatment of Hemophilia B.
Soroka Anastasiia B AB Feoktistova Sofya G SG Mityaeva Olga N ON Volchkov Pavel Y PY
International journal of molecular sciences 20230628 13
In contrast to the standard enzyme-replacement therapy, administered from once per 7-14 days to 2-3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain it for a prolonged time. In clinical research, the approach of delivering a functional copy of a gene using adeno-associated viral (AAV) vectors is widely used. The scientific community is actively researching possible modifications to improve delivery effi ...[more]
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