Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.

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Induced splice modulation of pre-mRNAs shows promise to correct aberrant disease transcripts and restore functional protein and thus has therapeutic potential. Duchenne muscular dystrophy (DMD) results from mutations that disrupt the DMD gene open reading frame causing an absence of dystrophin prote...

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Bibliographic Details
Main Authors:	Yin, H, Saleh, A, Betts, C, Camelliti, P, Seow, Y, Ashraf, S, Arzumanov, A, Hammond, S, Merritt, T, Gait, M, Wood, M
Format:	Journal article
Language:	English
Published:	2011

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