Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the HumanCLN2cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis
Author:
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.2004.15.1131
Reference46 articles.
1. A Phase I Study of Aerosolized Administration of tgAAVCF to Cystic Fibrosis Subjects with Mild Lung Disease
2. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
3. Retinal Degeneration in Retinitis Pigmentosa and Neuronal Ceroid Lipofuscinosis: An Overview
4. N-Acetyl-L-Aspartic acid: A literature review of a compound prominent in 1H-NMR spectroscopic studies of brain
Cited by 102 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. Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders;Advanced Drug Delivery Reviews;2023-06
2. A mouse mutant deficient in both neuronal ceroid lipofuscinosis‐associated proteins CLN3 and TPP1;Journal of Inherited Metabolic Disease;2023-04-26
3. AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges;Journal of Controlled Release;2023-03
4. Viral-mediated gene therapy in pediatric neurological disorders;World Journal of Pediatrics;2023-01-06
5. Aggregation chimeras provide evidence of in vivo intercellular correction in ovine CLN6 neuronal ceroid lipofuscinosis (Batten disease);PLOS ONE;2022-04-11
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