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Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines

ISCU myopathy is an inherited disease that primarily affects individuals of northern Swedish descent who share a single point mutation in the fourth intron of the ISCU gene. The current study shows correction of specific phenotypes associated with disease following treatment with an antisense oligon...

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Bibliografske podrobnosti
izdano v:Hum Mol Genet
Main Authors: Holmes-Hampton, Gregory P., Crooks, Daniel R., Haller, Ronald G., Guo, Shuling, Freier, Susan M., Monia, Brett P., Rouault, Tracey A.
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2016
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6078641/
https://ncbi.nlm.nih.gov/pubmed/28007899
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddw338
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