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

• Main Authors: Corinne Betts (Author), Amer F Saleh (Author), Andrey A Arzumanov (Author), Suzan M Hammond (Author), Caroline Godfrey (Author), Thibault Coursindel (Author), Michael J Gait (Author), Matthew JA Wood (Author)
• Format: Book
• Published: Elsevier, 2012-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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. C...