LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts
Touznik, Aleksander, Maruyama, Rika, Hosoki, Kana, Echigoya, Yusuke, Yokota, ToshifumiVolume:
7
Langue:
english
Journal:
Scientific Reports
DOI:
10.1038/s41598-017-03850-2
Date:
December, 2017
Fichier:
PDF, 4.97 MB
english, 2017