Title: | Structural basis of a small molecule targeting RNA for a specific splicing correction |
Journal : | Nature chemical biology |
Authors: | Campagne S, Author ; Boigner S ; Rudisser S ; Moursy A ; Gillioz L ; Knorlein A ; Hall J ; Ratni H ; Cléry A ; Allain FH |
Material Type: | Article |
Publisher: | United States, 10/2019 |
Languages: | English |
Keywords : | alternative splicing ; modes of action ; motor neuron disease ; neuromuscular disease ; RESEARCH ; small molecules ; SMN1-related proximal spinal muscular atrophy ; SMN2 gene ; spinal muscular atrophy |
Pubmed / DOI : | Pubmed : 31636429 / DOI : 10.1038/s41589-019-0384-5 |
N° Profil MNM : | 2019102 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31636429 |