Title: | Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy |
Journal : | Human molecular genetics, 25, 10 |
Authors: | Zhao X, Author ; Feng Z ; Ling KK ; Mollin A ; Sheedy J ; Yeh S ; Petruska J ; Narasimhan J ; Dakka A ; Welch EM ; Karp G ; Chen KS ; Metzger F ; Ratni H ; Lotti F ; Tisdale S ; Naryshkin NA ; Pellizzoni L ; Paushkin S ; Ko CP ; Weetall M |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 1885 |
Languages: | English |
Keywords : | alternative splicing ; cross-sectional study ; ephrin ; gene therapy ; pharmacodynamics ; pharmacokinetics ; post-transcriptional RNA processing ; SMN1-related proximal spinal muscular atrophy ; SMN2 gene ; snRNP ; spinal muscular atrophy mouse ; therapeutic prospects |
Pubmed / DOI : | DOI : 10.1093/hmg/ddw062 / Pubmed : 26931466 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/26931466 |