Title: | A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells |
Journal : | Cell stem cell, 18, 4 |
Authors: | Young CS, Author ; Hicks MR ; Ermolova NV ; Nakano H ; Jan M ; Younesi S ; Karumbayaram S ; Kumagai-Cresse C ; Wang D ; Zack JA ; Kohn DB ; Nakano A ; Nelson SF ; Miceli MC ; Spencer MJ ; Pyle AD |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 533 |
Languages: | English |
Keywords : | CRISPR/Cas9 ; Duchenne muscular dystrophy ; dystrophin ; exon skipping ; heart muscle fibers ; in vitro ; in vivo ; induced pluripotent stem cells ; myotube ; restriction enzyme ; sarcolemma |
Abstract: |
Comment in:
Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy. [Cell Stem Cell. 2016] |
Pubmed / DOI : | DOI : 10.1016/j.stem.2016.01.021 / Pubmed : 26877224 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/26877224 |