Gene Therapy Approaches for the Treatment of Hemophilia B


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 efficiency and expression. In preclinical studies, the possibility of genome editing using CRISPR/Cas9 technology for the treatment of hemophilia B is also being actively studied.

Authors Soroka AB, Feoktistova SG, Mityaeva ON, Volchkov PY
Journal International journal of molecular sciences
Publication Date 2023 Jun 28;24(13)
PubMed 37445943
PubMed Central PMC10341900
DOI 10.3390/ijms241310766

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