Optimization of trans-Splicing for Huntington's Disease RNA Therapy

Huntington's disease (HD) is a devastating neurodegenerative disorder caused by a polyglutamine (polyQ) expansion in exon 1 of the Huntingtin (HTT) gene. We have previously demonstrated that spliceosome-mediated trans-splicing is a viable molecular strategy to specifically reduce and repair mut...

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Udgivet i:Front Neurosci
Main Authors: Rindt, Hansjörg, Tom, Colton M., Lorson, Christian L., Mattis, Virginia B.
Format: Artigo
Sprog:Inglês
Udgivet: Frontiers Media S.A. 2017
Fag:
Neuroscience
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5641306/
https://ncbi.nlm.nih.gov/pubmed/29066943
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnins.2017.00544
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