Optimization of trans-Splicing for Huntington's Disease RNA Therapy
نویسندگان
چکیده
منابع مشابه
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 mutant HTT (mtHTT). Here, the targeted tethering efficacy of the pre-mRNA trans-splicing modules (PTM) in...
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ژورنال
عنوان ژورنال: Frontiers in Neuroscience
سال: 2017
ISSN: 1662-453X
DOI: 10.3389/fnins.2017.00544