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Therapies targeting DNA and RNA in Huntington s disease #MMPMID28920889
Wild EJ; Tabrizi S
Lancet Neurol 2017[Oct]; 16 (10): 837-47 PMID28920889show ga
No disease-slowing treatments exist for Huntington?s disease (HD), but its monogenic inheritance makes it an appealing candidate for the development of therapeutics targeting pathogenic processes close to its root genetic cause. HD is caused by CAG repeat expansions in the HTT gene, which encodes huntingtin protein; targeting of HTT transcription and the translation of its mRNA are therefore an area of intense investigation. ?Huntingtin-lowering? strategies include antisense oligonucleotides (ASOs) and RNA interference compounds targeting mRNA; and zinc-finger transcriptional repressors (ZFTR) and newer CRISPR/Cas9 methods aiming to reduce transcription by targeting DNA. An intrathecally-delivered ASO targeting total huntingtin is now well into its first human clinical trial, with other ASO approaches expected to enter trials within the next 1-2 years, and virally-delivered RNAi and ZFTR in advanced testing in animal models. Meanwhile, recent advances to improve the design and delivery of targeted therapeutics are likely to improve their efficacy, safety, tolerability and duration of effect.