Title: | Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model |
Journal : | Science advance, 1, 2 |
Authors: | Nizzardo M, Author ; Simone C ; Rizzo F ; Salani S ; Dametti S ; Rinchetti P ; Del Bo R ; Foust K ; Kaspar BK ; Bresolin N ; Comi GP ; Corti S |
Material Type: | Article |
Publication Date: | 2015 |
Languages: | English |
Keywords : | AAV9 vectors ; cell cultures ; cell expression ; cross-sectional study ; embryonic stem cell ; gene therapy ; IGHMBP2 gene ; immunohistochemistry ; motor neurons ; mouse model ; spinal muscular atrophy with respiratory distress type 1 |
Keywords: | Spinal Muscular atrophy with Respiratory Distress Type 1;gene therapy; |
Pubmed / DOI : | DOI : 10.1126/sciadv.1500078 / Pubmed : 26601156 |
See also : |