Title: | Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy |
Journal : | Molecular therapy. Nucleic acids, 10 |
Authors: | Azibani F, Author ; Brull A ; Arandel L ; Beuvin M ; Nelson I ; Jollet A ; Ziat E ; Prudhon B ; Benkhelifa Ziyyat S ; Bitoun M ; Lorain S ; Bonne G ; Bertrand AT |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 376 |
Languages: | English |
Keywords : | adenoassociated vectors ; gene therapy ; lamin A/C ; LMNA gene ; LMNA-related congenital muscular dystrophhy ; messenger RNA ; mouse model ; myoblast ; myotube ; splicing ; therapeutic prospects ; trans-splicing |
Keywords: | LMNA-related congenital muscular dystrophy;Lmna;gene therapy;lamin A/C;trans-splicing; |
Pubmed / DOI : | DOI : 10.1016/j.omtn.2017.12.012 / Pubmed : 29499949 |
N° Profil MNM : | 2018031 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29499949 |