Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

Journal article

Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. Challenges in the development of this approach are the relatively poor systemic AO delivery and inefficient dystrophin correction in affected non-skeletal muscle tissues, including the heart. We hav...

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Authors: Betts, C; Saleh, A; Arzumanov, A; Hammond, S; Godfrey, C

• Publication status: Published
• Journal: Molecular therapy. Nucleic acids
• Volume: 1
• Issue: 8
• Pages: e38
• Publication date: 2012-01-01
• DOI: 10.1038/mtna.2012.30
• EISSN: 2162-2531
• ISSN: 2162-2531
• Language: English
• Keywords: Cardiac muscle; Antisense oligonucleotide; Duchenne muscular dystrophy; Phosphorodiamidate morpholino oligomer; PMO internalizing peptide