Formulation and delivery of splice-correction antisense oligonucleotides by amino acid modified polyethylenimine.

Abstract

Splice-correcting phosphorothioate RNA antisense oligonucleotides with 2'-O-methyl modifications (ASO) are promising therapeutic agents for several disorders caused by aberrant splicing. However, their usefulness is hindered by the lack of efficient delivery. Unmodified 25 kDa polyethylenimine (PEI) has shown potential for plasmid delivery but seems to be… (More)
DOI: 10.1021/mp900220p

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