In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

BACKGROUND: Targeted splice modulation of pre-mRNA transcripts by antisense oligonucleotides (AOs) can correct the function of aberrant disease-related genes. Duchenne muscular dystrophy (DMD) arises as a result of mutations that interrupt the open-reading frame in the DMD gene encoding dystrophin s...

書誌詳細
主要な著者:	Wang, Q, Yin, H, Camelliti, P, Betts, C, Moulton, H, Lee, H, Saleh, A, Gait, M, Wood, M
フォーマット:	Journal article
言語:	English
出版事項:	2010

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