A blood-brain-barrier penetrant AAV gene therapy improves neurologic function in symptomatic mucolipidosis IV mice.

Madison Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, S. Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk
{"title":"A blood-brain-barrier penetrant AAV gene therapy improves neurologic function in symptomatic mucolipidosis IV mice.","authors":"Madison Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, S. Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk","doi":"10.1016/j.omtm.2024.101269","DOIUrl":null,"url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"59 3","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Therapy - Methods & Clinical Development","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.omtm.2024.101269","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
血脑屏障穿透型 AAV 基因疗法可改善症状性粘脂病 IV 小鼠的神经功能。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
自引率
0.00%
发文量
0
期刊最新文献
Evaluation of a Rapid Multi-Attribute Combinatorial High-Throughput UV-Vis/DLS/SLS Analytical Platform for rAAV Quantification and Characterization Biomanufacturing in gene and cell therapy Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells Mesenchymal Stromal Cells Protect Combined Oncolytic and Helper Dependent Adenoviruses from Humoral Immunity
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1