A Prospective Study in the Rational Design of Efficient Antisense Oligonucleotides for Exon Skipping in the DMD Gene

Antisense oligonucleotide (AON)-mediated exon skipping to restore dystrophin expression in Duchenne muscular dystrophy (DMD) therapy shown promise in a number of human clinical trials. Current AON design methods are semi-empirical, involving either trial-and-error and/or preliminary experimentations...

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Detalhes bibliográficos
Main Authors: Pramono, Zacharias Aloysius Dwi, Wee, Keng Boon, Wang, Jian Li, Chen, Yi Jun, Xiong, Qian Bin, Lai, Poh San, Yee, Woon Chee
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2012
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Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3404420/
https://ncbi.nlm.nih.gov/pubmed/22486275
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.205
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