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 approa...

Descripción completa

Detalles Bibliográficos
Autores principales: Soroka, Anastasiia B., Feoktistova, Sofya G., Mityaeva, Olga N., Volchkov, Pavel Y.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10341900/
https://www.ncbi.nlm.nih.gov/pubmed/37445943
http://dx.doi.org/10.3390/ijms241310766
_version_ 1785072371950944256
author Soroka, Anastasiia B.
Feoktistova, Sofya G.
Mityaeva, Olga N.
Volchkov, Pavel Y.
author_facet Soroka, Anastasiia B.
Feoktistova, Sofya G.
Mityaeva, Olga N.
Volchkov, Pavel Y.
author_sort Soroka, Anastasiia B.
collection PubMed
description 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.
format Online
Article
Text
id pubmed-10341900
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-103419002023-07-14 Gene Therapy Approaches for the Treatment of Hemophilia B Soroka, Anastasiia B. Feoktistova, Sofya G. Mityaeva, Olga N. Volchkov, Pavel Y. Int J Mol Sci Review 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. MDPI 2023-06-28 /pmc/articles/PMC10341900/ /pubmed/37445943 http://dx.doi.org/10.3390/ijms241310766 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Soroka, Anastasiia B.
Feoktistova, Sofya G.
Mityaeva, Olga N.
Volchkov, Pavel Y.
Gene Therapy Approaches for the Treatment of Hemophilia B
title Gene Therapy Approaches for the Treatment of Hemophilia B
title_full Gene Therapy Approaches for the Treatment of Hemophilia B
title_fullStr Gene Therapy Approaches for the Treatment of Hemophilia B
title_full_unstemmed Gene Therapy Approaches for the Treatment of Hemophilia B
title_short Gene Therapy Approaches for the Treatment of Hemophilia B
title_sort gene therapy approaches for the treatment of hemophilia b
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10341900/
https://www.ncbi.nlm.nih.gov/pubmed/37445943
http://dx.doi.org/10.3390/ijms241310766
work_keys_str_mv AT sorokaanastasiiab genetherapyapproachesforthetreatmentofhemophiliab
AT feoktistovasofyag genetherapyapproachesforthetreatmentofhemophiliab
AT mityaevaolgan genetherapyapproachesforthetreatmentofhemophiliab
AT volchkovpavely genetherapyapproachesforthetreatmentofhemophiliab

Ejemplares similares