Huntingtin Lowering Strategies

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Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.

The idea to lower mutant huntingtin is especially appealing in Huntington's disease (HD). It is autosomal dominant, so that expression of the mutant allele causes the disease. Advances in RNA and gene regulation provide foundations for the huntingtin gene (both normal and mutant alleles) and possibly the mutant allele only. There is much preclinical animal work to support the concept of gene an...

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ژورنال

عنوان ژورنال: International Journal of Molecular Sciences

سال: 2020

ISSN: 1422-0067

DOI: 10.3390/ijms21062146