van DeutekomJC, JansonAA, GinjaarIB, et al.Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med, 2007; 357:2677–2686.
2.
KinaliM, Arechavala-GomezaV, FengL, et al.Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol, 2009; 8:918–928.
3.
SchumperliD, PillaiRS. The special Sm core structure of the U7 snRNP: far-reaching significance of a small nuclear ribonucleoprotein. Cell Mol Life Sci, 2004; 61:2560–2570.
4.
GoyenvalleA, VulinA, FougeroussF, et al.Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science, 2004; 306:1796–1799.
5.
GoyenvalleA, WrightJ, BabbsA, et al.Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy. Mol Ther, 2012; 20:1212–1221.
6.
WeinN, DunnDM, WaldropMA, et al.Absence of significant off-target splicing variation with a U7snRNA vector targeting DMD exon 2 duplications. Hum Gene Ther, 2021; 32:1346–1359.