Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

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Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure with drisapersen, an AO candidate drug in phase 3 trial, highlights the importance of exploring other effective AO chemistries for DMD. Previ...

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Detalles Bibliográficos
Autores principales: Gao, Xianjun, Shen, Xiaoyong, Dong, Xue, Ran, Ning, Han, Gang, Cao, Limin, Gu, Ben, Yin, HaiFang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4881755/
https://www.ncbi.nlm.nih.gov/pubmed/26440599
http://dx.doi.org/10.1038/mtna.2015.27
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