Title: | A gene-edited mouse model of Limb-Girdle muscular dystrophy 2C for testing exon skipping |
Journal : | Disease models & mechanisms |
Authors: | Demonbreun AR, Author ; Wyatt EJ ; Fallon KS ; Oosterbaan CC ; Page P ; Hadhazy M ; Quattrocelli M ; Barefield DY ; McNally EM |
Material Type: | Article |
Publisher: | England, 10/2019 |
Languages: | English |
Keywords : | CRISPR/Cas9 ; gamma-sarcoglycan-related limb-girdle muscular dystrophy R5 ; gene therapy ; limb girdle muscular dystrophy ; mouse model ; neuromuscular disease ; RESEARCH |
Keywords: | antisense oligonucleotide;dystrophin;exon skipping;gene correction;limb girdle;muscular dystrophy;sarcoglycan; |
Pubmed / DOI : | Pubmed : 31582396 / DOI : 10.1242/dmm.040832 |
N° Profil MNM : | 2019101 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31582396 |