Oligonucleotide therapeutic approaches for Huntington disease
نویسندگان
چکیده
منابع مشابه
Oligonucleotide therapeutic approaches for Huntington disease.
Huntington disease is an autosomal dominant neurodegenerative disorder caused by a toxic expansion in the CAG repeat region of the huntingtin gene. Oligonucleotide approaches based on RNAi and antisense oligonucleotides provide promising new therapeutic strategies for direct intervention through reduced production of the causative mutant protein. Allele-specific and simultaneous mutant and wild...
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ژورنال
عنوان ژورنال: Journal of Clinical Investigation
سال: 2011
ISSN: 0021-9738
DOI: 10.1172/jci45130