Title : Therapies targeting DNA and RNA in Huntington's disease.

Pub. Date : 2017 Oct

PMID : 28920889






2 Functional Relationships(s)
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Protein Name
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1 Huntingtin-lowering strategies include antisense oligonucleotides and RNA interference targeting mRNA, and zinc finger transcriptional repressors and CRISPR-Cas9 methods aiming to reduce transcription by targeting DNA. Oligonucleotides huntingtin Homo sapiens
2 An intrathecally delivered antisense oligonucleotide that aims to lower huntingtin is now well into its first human clinical trial, with other antisense oligonucleotides expected to enter trials in the next 1-2 years and virally delivered RNA interference and zinc finger transcriptional repressors in advanced testing in animal models. Oligonucleotides huntingtin Homo sapiens