UCL logo

UCL Discovery

UCL home » Library Services » Electronic resources » UCL Discovery

Therapies targeting DNA and RNA in Huntington's disease

Wild, EJ; Tabrizi, SJ; (2017) Therapies targeting DNA and RNA in Huntington's disease. The Lancet Neurology , 16 (10) pp. 837-847. 10.1016/S1474-4422(17)30280-6. Green open access

Wild_FINAL HD_therapeutics_review_R2-clean.pdf - ["content_typename_Accepted version" not defined]

Download (700kB) | Preview


No disease-slowing treatment exists for Huntington's disease, but its monogenic inheritance makes it an appealing candidate for the development of therapies targeting processes close to its genetic cause. Huntington's disease is caused by CAG repeat expansions in the HTT gene, which encodes the huntingtin protein; development of therapies to target HTT transcription and the translation of its mRNA is therefore an area of intense investigation. 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. 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. Recent advances in the design and delivery of therapies to target HTT RNA and DNA are expected to improve their efficacy, safety, tolerability, and duration of effect in future studies.

Type: Article
Title: Therapies targeting DNA and RNA in Huntington's disease
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/S1474-4422(17)30280-6
Publisher version: http://dx.doi.org/10.1016/S1474-4422(17)30280-6
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
URI: http://discovery.ucl.ac.uk/id/eprint/1563763
Downloads since deposit
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item