Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia
Author:
Funder
Natural Science Foundation of Zhejiang Province (CN)
National Natural Science Foundation of China
Publisher
Public Library of Science (PLoS)
Subject
Multidisciplinary
Reference52 articles.
1. Mutations in the cone photoreceptor G-protein alpha-subunit gene GNAT2 in patients with achromatopsia;S Kohl;Am J Hum Genet,2002
2. Effect of gene therapy on visual function in Leber's congenital amaurosis;JW Bainbridge;N Engl J Med,2008
3. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics;AV Cideciyan;Proc Natl Acad Sci U S A,2008
4. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial;AM Maguire;Lancet,2009
5. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year;AV Cideciyan;Hum Gene Ther,2009
Cited by 15 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. A novel capsid-XL32-derived adeno-associated virus serotype prompts retinal tropism and ameliorates choroidal neovascularization;Biomaterials;2024-01
2. Phenotype and genotype of 15 Saudi patients with achromatopsia: A case series;Saudi Journal of Ophthalmology;2023-09-16
3. Gene therapy for inherited retinal disease: long-term durability of effect;Ophthalmic Research;2022-09-14
4. CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa;Molecular Therapy;2022-04
5. Delineating the Molecular and Phenotypic Spectrum of the CNGA3-Related Cone Photoreceptor Disorder in Pakistani Families;Genes;2022-03-29
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