Ontology highlight
ABSTRACT:
SUBMITTER: Dastidar S
PROVIDER: S-EPMC6144820 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Dastidar Sumitava S Ardui Simon S Singh Kshitiz K Majumdar Debanjana D Nair Nisha N Fu Yanfang Y Reyon Deepak D Samara Ermira E Gerli Mattia F M MFM Klein Arnaud F AF De Schrijver Wito W Tipanee Jaitip J Seneca Sara S Tulalamba Warut W Wang Hui H Chai Yoke Chin YC In't Veld Peter P Furling Denis D Tedesco Francesco Saverio FS Vermeesch Joris R JR Joung J Keith JK Chuah Marinee K MK VandenDriessche Thierry T
Nucleic acids research 20180901 16
CRISPR/Cas9 is an attractive platform to potentially correct dominant genetic diseases by gene editing with unprecedented precision. In the current proof-of-principle study, we explored the use of CRISPR/Cas9 for gene-editing in myotonic dystrophy type-1 (DM1), an autosomal-dominant muscle disorder, by excising the CTG-repeat expansion in the 3'-untranslated-region (UTR) of the human myotonic dystrophy protein kinase (DMPK) gene in DM1 patient-specific induced pluripotent stem cells (DM1-iPSC), ...[more]