GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells

出版年份 2017 全文链接

标题

作者

关键词

myogenic progenitors, induced pluripotent stem cell, antisense oligonucleotide, Pompe disease, IVS1 mutation, skeletal muscle differentiation, pre-mRNA splicing, pseudo exon, exon inclusion, lysosomal storage disorder

出版物

Molecular Therapy-Nucleic Acids

Volume 7, Issue -, Pages 101-115

出版商

Elsevier BV

发表日期

2017-03-14

DOI

10.1016/j.omtn.2017.03.002

向作者/读者发起求助以获取更多资源

Protocol

在线求助

Reagent

在线求助

Reprint

联系作者

Discover Peeref hubs

Discuss science. Find collaborators. Network.

Join a conversation

Create your own webinar

Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.

Create Now