Gene Therapy Approaches for the Treatment of Hemophilia B.
Anastasiia B SorokaSofya G FeoktistovaOlga N MityaevaPavel Y VolchkovPublished in: International journal of molecular sciences (2023)
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.
Keyphrases
- gene therapy
- crispr cas
- genome editing
- replacement therapy
- gene expression
- healthcare
- magnetic resonance
- poor prognosis
- mental health
- dna methylation
- sars cov
- randomized controlled trial
- stem cells
- clinical trial
- early onset
- magnetic resonance imaging
- computed tomography
- copy number
- long non coding rna
- bone marrow
- binding protein
- genome wide
- contrast enhanced
- double blind
- genome wide analysis