基因治疗血友病 B 的方法。
Gene Therapy Approaches for the Treatment of Hemophilia B.
机构信息
Life Sciences Research Center, Moscow Institute of Physics and Technology, National Research University, 141700 Dolgoprudniy, Russia.
出版信息
Int J Mol Sci. 2023 Jun 28;24(13):10766. doi: 10.3390/ijms241310766.
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.
摘要
与标准的酶替代疗法不同，重症乙型血友病患者每 7-14 天接受一次治疗，改为每周 2-3 次，基因治疗可以单次注射恢复 F9 基因表达，并长时间维持。在临床研究中，使用腺相关病毒（AAV）载体传递功能基因副本的方法被广泛应用。科学界正在积极研究可能的修饰方法以提高传递效率和表达水平。在临床前研究中，使用 CRISPR/Cas9 技术进行基因组编辑治疗乙型血友病的可能性也在积极研究中。
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