Gene Therapy Approaches for the Treatment of Hemophilia B
Anastasiia B. Soroka, Sofya G. Feoktistova, Olga Mityaeva, Pavel Volchkov
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.
Topics & Concepts
Genetic enhancementCRISPRGenome editingFactor IXGeneCas9Gene deliveryBioinformaticsGene expressionViral vectorMedicineComputational biologyGenomeBiologyGeneticsRecombinant DNACRISPR and Genetic EngineeringVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects