Gene Therapy Approaches for the Treatment of Hemophilia B.
AAV
blood coagulation factor IX
gene therapy
genome editing
hemophilia B
hemostasis
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
28 Jun 2023
28 Jun 2023
Historique:
received:
15
05
2023
revised:
20
06
2023
accepted:
26
06
2023
medline:
17
7
2023
pubmed:
14
7
2023
entrez:
14
7
2023
Statut:
epublish
Résumé
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.
Identifiants
pubmed: 37445943
pii: ijms241310766
doi: 10.3390/ijms241310766
pmc: PMC10341900
pii:
doi:
Substances chimiques
Factor IX
9001-28-9
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Russian Science Foundation
ID : 23-64-00002
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