Title: | Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice |
Journal : | The Journal of clinical investigation |
Authors: | Klein AF ; Varela MA ; Arandel L ; Holland A ; Naouar N ; Arzumanov A ; Seoane D ; Revillod L ; Bassez G ; Ferry A ; Jauvin D ; Gourdon G ; Puymirat J ; Gait MJ ; Furling D ; Wood MJA |
Material Type: | Article |
Publisher: | United States, 09/2019 |
Languages: | English |
Keywords : | antisense oligonucleotides ; glycoproteins ; myotonic dystrophy type 1 ; neuromuscular disease ; RESEARCH |
Keywords: | Genetic diseases;Muscle;Therapeutics; |
Pubmed / DOI : | Pubmed : 31479430 / DOI : 10.1172/JCI128205 |
N° Profil MNM : | 2019092 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31479430 |
See also : |