Title: | Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy |
Journal : | Cell, 185, 12 |
Authors: | Marasco LE ; Dujardin G ; Sousa Luis R ; Liu YH ; Stigliano JN ; Nomakuchi T ; Proudfoot NJ ; Krainer AR ; Kornblihtt AR |
Material Type: | Article |
Publisher: | United States, 09/06/2022 |
Size: | p 2057 |
Languages: | English |
Keywords : | antisense oligonucleotides ; histone deacetylase inhibitors ; mouse model ; nusinersen ; RESEARCH ; spinal muscular atrophy |
Keywords: | alternative splicing;antisense oligonucleotide therapy;chromatin;histone acetylation;spinal muscular atrophy;transcriptional elongation; |
Pubmed / DOI : | Pubmed : 35688133 / DOI : 10.1016/j.cell.2022.04.031 |
N° Profil MNM : | 2022061 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/35688133 |