Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

survival of motor neuron 2, centromeric (SMN2) is a gene that modifies the severity of spinal muscular atrophy (SMA), a motor-neuron disease that is the leading genetic cause of infant mortality. Increasing inclusion of SMN2 exon 7, which is predominantly skipped, holds promise to treat or possibly...

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Bibliographic Details
Main Authors: Hua, Yimin, Vickers, Timothy A., Okunola, Hazeem L., Bennett, C. Frank, Krainer, Adrian R.
Format: Artigo
Language:Inglês
Published: American Society of Human Genetics 2008
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2427210/
https://ncbi.nlm.nih.gov/pubmed/18371932
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2008.01.014
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