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

• Asıl Yazarlar: Corinne Betts (Yazar), Amer F Saleh (Yazar), Andrey A Arzumanov (Yazar), Suzan M Hammond (Yazar), Caroline Godfrey (Yazar), Thibault Coursindel (Yazar), Michael J Gait (Yazar), Matthew JA Wood (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Elsevier, 2012-01-01T00:00:00Z.
• Konular: article
• Online Erişim: 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...