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...

Descripción completa

Detalles Bibliográficos
Autores principales: Betts, Corinne, Saleh, Amer F, Arzumanov, Andrey A, Hammond, Suzan M, Godfrey, Caroline, Coursindel, Thibault, Gait, Michael J, Wood, Matthew JA
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2012
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3438601/
https://www.ncbi.nlm.nih.gov/pubmed/23344180
http://dx.doi.org/10.1038/mtna.2012.30

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3438601/
https://www.ncbi.nlm.nih.gov/pubmed/23344180
http://dx.doi.org/10.1038/mtna.2012.30

Ejemplares similares