TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals

Antisense oligonucleotides (ASOs) are versatile molecules that can be designed to specifically alter splicing patterns of target pre-mRNAs. Here we exploit this feature to phenocopy a genetic disease. Spinal muscular atrophy (SMA) is a motor neuron disease caused by loss-of-function mutations in the...

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Bibliografiske detaljer
Main Authors: Sahashi, Kentaro, Hua, Yimin, Ling, Karen K.Y., Hung, Gene, Rigo, Frank, Horev, Guy, Katsuno, Masahisa, Sobue, Gen, Ko, Chien-Ping, Bennett, C. Frank, Krainer, Adrian R.
Format: Artigo
Sprog:Inglês
Udgivet: Cold Spring Harbor Laboratory Press 2012
Fag:
Resource/Methodology
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3426765/
https://ncbi.nlm.nih.gov/pubmed/22895255
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/gad.197418.112
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