Title: | SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy |
Journal : | Science (New York, N.Y.), 345, 6197 |
Authors: | Naryshkin NA ; Weetall M ; Dakka A ; Narasimhan J ; Zhao X ; Feng Z ; Ling KK ; Karp GM ; Qi H ; Woll MG ; Chen G ; Zhang N ; Gabbeta V ; Vazirani P ; Bhattacharyya A ; Furia B ; Risher N ; Sheedy J ; Kong R ; Ma J ; Turpoff A ; Lee CS ; Zhang X ; Moon YC ; Trifillis P ; Welch EM ; Colacino JM ; Babiak J ; Almstead NG ; Peltz SW ; Eng LA ; Chen KS ; Mull JL ; Lynes MS ; Rubin LL ; Fontoura P ; Santarelli L ; Haehnke D ; McCarthy KD ; Schmucki R ; Ebeling M ; Sivaramakrishnan M ; Ko CP ; Paushkin SV ; Ratni H ; Gerlach I ; Ghosh A ; Metzger F |
Material Type: | Article |
Publication Date: | 2014 |
Size: | p 688 |
Languages: | English |
Keywords : | alternative splicing ; cross-sectional study ; gene therapy ; life expectancy ; messenger RNA ; molecular screening ; SMN protein ; SMN2 gene ; spinal muscular atrophy ; spinal muscular atrophy mouse ; therapeutic prospects |
Abstract: |
Comment in:
Molecular biology. A splicing magic bullet. Vigevani L, Valcárcel J. Science. 2014 Aug 8;345(6197):624-5. Neuromuscular disorders: Beefing up the right splice variant to treat spinal muscular atrophy. Cully M. Nat Rev Drug Discov. 2014 Oct;13(10):725. |
Pubmed / DOI : | DOI : 10.1126/science.1250127 / Pubmed : 25104390 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/25104390 |
See also : |