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Browsing by Author "Rabinowitz, Adam"
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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Alter, Julia; Lou, Fang; Rabinowitz, Adam; Yin, HaiFang; Rosenfeld, Jeffrey; Wilton, Steve D; Partridge, Terence A; Lu, Qi Long (2006)For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of ...