Audacity of gene therapy

被引：0

作者：

Dimitrov, Marketa ^{[1
]}

Ebens, Christen L. ^{[1
]}

Tolar, Jakub ^{[1
]}

机构：

[1] Univ Minnesota, Dept Paediat, Minneapolis, MN 55455 USA

来源：

BRITISH JOURNAL OF DERMATOLOGY | 2024年 / 191卷 / 06期

关键词：

D O I：

10.1093/bjd/ljae332

中图分类号：

R75 [皮肤病学与性病学];

学科分类号：

100206 ;

摘要：

Since the identification of DNA as the hereditary material, therapeutic advances have focused on gene therapy and allogeneic haematopoietic cell transplants for inherited diseases. Despite successes in treating conditions like beta -thalassaemia and sickle cell disease, challenges such as insertional oncogenesis and immunogenicity persist. Future efforts must prioritize improving vector safety, refining manufacturing processes and expanding access to gene therapy, to fully realize its potential.

引用

页码：1009 / 1011

页数：3

共 8 条

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