Gene Therapy Approaches for the Treatment of Hemophilia B.

Article Details

Citation
Copy to clipboard

Soroka AB, Feoktistova SG, Mityaeva ON, Volchkov PY

Gene Therapy Approaches for the Treatment of Hemophilia B.

Int J Mol Sci. 2023 Jun 28;24(13):10766. doi: 10.3390/ijms241310766.

PubMed ID
37445943 [ View in PubMed
]
Abstract

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.

DrugBank Data that Cites this Article

Drugs