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

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...

Täydet tiedot

Bibliografiset tiedot
Päätekijät: Betts, C, Saleh, A, Arzumanov, A, Hammond, S, Godfrey, C, Coursindel, T, Gait, M, Wood, M
Aineistotyyppi: Journal article
Kieli:English
Julkaistu: 2012