Wilhelmus Wenceslaus Matthias PIJNAPPEL,Antje Tjitske VAN DER PLOEG,Erik VAN DER WAL,Jur KRUIJT,Pablo HERRERO HERNANDEZ
申请号：
US16645561
公开号：
US20200276334A1
申请日:
2018.09.07
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The invention relates to methods for gene therapy in a subject suffering from Pompe disease, comprising gene-editing of a glucosidase, acid, alpha gene (GAA) in said subject. The invention further relates to a cell culture of genetically changed, differentiated myogenic progenitor cells derived from a donor subject suffering from Pompe disease, to a vector for use in a method for gene-editing a eukaryotic cell, and to a myotube prepared from myogenic progenitor cells that have been genetically changed by gene-editing.
