Title: | Specific correction of alternative survival motor neuron 2 (SMN2) splicing by small molecules: Discovery of a potential novel medicine to treat spinal muscular atrophy |
Journal : | Journal of Medicinal Chemistry, 59, 13 |
Authors: | Ratni H, Author ; Karp GM ; Weetall M ; Naryshkin NN ; Paushkin SV ; Chen KS ; McCarthy KD ; Qi H ; Turpoff A ; Woll MG ; Zhang X ; Zhang N ; Yang T ; Dakka A ; Vazirani P ; Zhao X ; Pinard E ; Green L ; David-Pierson P ; Tuerck D ; Poirier A ; Muster W ; Kirchner S ; Mueller L ; Gerlach I ; Metzger F |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 6086 |
Languages: | English |
Keywords : | alternative splicing ; coumarin ; cross-sectional study ; gene therapy ; pharmacokinetics ; pharmacy ; RG7800 ; SMN protein ; SMN1-related proximal spinal muscular atrophy ; SMN2 gene ; therapeutic efficiency |
Pubmed / DOI : | DOI : 10.1021/acs.jmedchem.6b00459 / Pubmed : 27299419 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/27299419 |