Title: | Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy |
Journal : | Science translational medicine, 6, 220 |
Authors: | Childers MK ; Joubert R ; Poulard K ; Moal C ; Grange RW ; Doering JA ; Lawlor MW ; Rider BE ; Jamet T ; Daniele N ; Martin S ; Riviere C ; Soker T ; Hammer C ; Van Wittenberghe L ; Lockard M ; Guan X ; Goddard M ; Mitchell E ; Barber J ; Williams JK ; Mack DL ; Furth ME ; Vignaud A ; Masurier C ; Mavilio F ; Moullier P ; Beggs AH ; Buj Bello A |
Material Type: | Article |
Publication Date: | 2014 |
Languages: | English |
Keywords : | adenoassociated vectors ; cross-sectional study ; gene therapy ; immunofluorescence ; intravenous injection ; model dog ; mouse model ; MTM1 gene ; muscle histopathology ; muscle strength ; myotubularin ; X-linked myotubular myopathy |
Pubmed / DOI : | DOI : 10.1126/scitranslmed.3007523 / Pubmed : 24452262 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/24452262 |
See also : |