Title: | Autologous intramuscular transplantation of engineered satellite cells induces exosome-mediated systemic expression of Fukutin-related protein and rescues disease phenotype in a murine model of limb-girdle muscular dystrophy type 2I |
Journal : | Human molecular genetics, 26, 19 |
Authors: | Frattini P, Author ; Villa C ; de Santis F ; Meregalli M ; Belicchi M ; Erratico S ; Bella P ; Raimondi MT ; Lu Q ; Torrente Y |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 3682 |
Languages: | English |
Keywords : | alpha-dystroglycan ; alpha-dystroglycanopathy ; cell lines ; cell therapy ; exosome ; FKRP gene ; FKRP protein ; FKRP-related limb-girdle muscular dystrophy R9 ; glycosyltransferases ; hypertrophic progressive congenital muscular dystrophy ; intramuscular injection ; mouse model ; myoblast ; stem cells |
Pubmed / DOI : | DOI : 10.1093/hmg/ddx252 / Pubmed : 28666318 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28666318 |