Title: | Becker muscular dystrophy patients with deletions around exon 51 ; a promising outlook for exon skipping therapy in Duchenne patients |
Journal : | Neuromuscular disorders : NMD, 20, 4 |
Authors: | Helderman-van den Enden ATJM ; Straathof CSM ; Aartsma Rus A ; den Dunnen JT ; Verbist BM ; Bakker E ; Verschuuren JJGM ; Ginjaar HB |
Material Type: | Article |
Publication Date: | 2010 |
Size: | p. 251-254 |
Languages: | English |
Keywords : | Becker muscular dystrophy ; case studies ; Duchenne muscular dystrophy ; DYS gene ; dystrophin ; exon ; exon skipping ; gene therapy ; genetic deletions ; genetic mutations ; MRI ; phenotypes ; skeletal muscle |
Abstract: |
Accès au résumé PubMed / to PubMed abstract |
Lien associé : | http://www.ncbi.nlm.nih.gov/pubmed/20153965 |
Pubmed / DOI : | DOI : 10.1016/j.nmd.2010.01.013 |