Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

Antisense oligonucleotides (AOs) have the capacity to alter the processing of pre-mRNA transcripts in order to correct the function of aberrant disease-related genes. Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle degenerative disease that arises from mutations in the DMD gene leading...

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Detalhes bibliográficos
Main Authors: Yin, HaiFang, Betts, Corinne, Saleh, Amer F, Ivanova, Gabriela D, Lee, Hyunil, Seow, Yiqi, Kim, Dalsoo, Gait, Michael J, Wood, Matthew JA
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2010
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Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2848838/
https://ncbi.nlm.nih.gov/pubmed/20068555
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.310
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