Gene Therapy for Metachromatic Leukodystrophy; MLD
Author:
Affiliation:
1. Department of Biochemistry and Molecular Biology, Nippon Medical School
Publisher
Medical Association of Nippon Medical School
Subject
General Earth and Planetary Sciences,General Environmental Science
Link
https://www.jstage.jst.go.jp/article/manms/19/3/19_224/_pdf
Reference17 articles.
1. 1. Gomez-Ospina N: in GeneReviews ((R)) (M. P. Adam et al., eds) 1993.
2. 2. I’ Dali C. Sevin C. Krägeloh, et al.: Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial. Mol Genet Metab 2020; 131: 235-244. doi: 10.1016/j.ymgme.2020.07.002.
3. 3. Rosenberg JB, Sondhi D, Rubin DG, et al.: Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates. Hum Gene Ther Clin Dev 2014; 25: 164-177. doi: 10.1089/humc.2013.239.
4. 4. Piguet F, Sondhi D, Piraud M, et al.: Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. Hum Gene Ther 2012; 23: 903-914. doi: 10.1089/hum.2012.015.
5. 5. Sevin C, Roujeau T, Cartier N, et al.: Intracerebral gene therapy in children with metachromatic leukodystrophy. Mol Genet Metab 2018; 123: S129. doi: 10.1016/j.ymgme.2017.12.352.
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