Title: | Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy |
Journal : | Human genetics, 135, 9 |
Authors: | Robinson-Hamm JN, Author ; Gersbach CA |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 1029 |
Languages: | English |
Keywords : | antisense oligonucleotides ; CRISPR/Cas9 ; Duchenne muscular dystrophy ; DYS gene ; exon skipping ; gene therapy ; genome editing ; minidystrophin ; review article ; therapeutic prospects |
Pubmed / DOI : | DOI : 10.1007/s00439-016-1725-z / Pubmed : 27542949 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/27542949 |