Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Physics and Astronomy,General Biochemistry, Genetics and Molecular Biology,General Chemistry
Link
http://www.nature.com/articles/s41467-020-19507-0.pdf
Reference9 articles.
1. Russell, S. et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in subjects with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open label phase 3 trial. Lancet 390, 849–860 (2017).
2. Mendell, J. R. et al. Single dose gene replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017).
3. Chung, D. C. et al. A novel mobility test to assess functional vision in patients with inherited retinal dystrophies. Clin. Exp. Ophthalmol. 46, 247–259 (2018).
4. Mingozzi, F. & High, K. A. Overcoming the host immune response to AAV gene delivery vectors: the race between clearance, tolerance, neutralization, and escape. Annual Rev. Virol. 4, 511–534 (2017).
5. Boyce, N. Trial halted after gene shows up in semen. Nature 414, 677 (2001).
Cited by 22 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art;Bleeding, Thrombosis and Vascular Biology;2024-09-04
2. Somatic Gene Therapy: Ethics and Access;Annual Review of Genomics and Human Genetics;2024-08-27
3. Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine;Genes & Diseases;2024-01
4. Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease;Nature Medicine;2023-11-16
5. IRSF 2023 - Rett Syndrome Scientific Meeting Report;Translational Science of Rare Diseases;2023-09-30
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3