期刊
JOURNAL OF CENTRAL NERVOUS SYSTEM DISEASE
卷 14, 期 -, 页码 -出版社
SAGE PUBLICATIONS LTD
DOI: 10.1177/11795735221092517
关键词
chorea; antipsychotic medication; antidepressants; mood stabilizers; antisense oligonucleotides; RNAi therapies; Huntington's disease
资金
- Oakley Mental Health Research Foundation [3725204]
- Alzheimers New Zealand Charitable Trust [370836]
- Alzheimers New Zealand [3718869]
- Freemasons New Zealand [3719321]
- Aotearoa Foundation, Centre for Brain Research and University of Auckland [3705579]
- Neurological Foundation of New Zealand [848010]
- Brain Research New Zealand
- Health Research Council of New Zealand [3627373]
Huntington's disease (HD) is a neurodegenerative disease with no cure. Current treatments focus on managing symptoms, while emerging therapies aim to lower levels of mutant huntingtin protein. These include antisense oligonucleotide therapies, RNA interference therapies, and stem cell therapies, among others. Additionally, there are potential therapies in pre-clinical development.
Huntington's disease (HD) is an autosomal neurodegenerative disease that is characterized by an excessive number of CAG trinucleotide repeats within the huntingtin gene (HTT). HD patients can present with a variety of symptoms including chorea, behavioural and psychiatric abnormalities and cognitive decline. Each patient has a unique combination of symptoms, and although these can be managed using a range of medications and non-drug treatments there is currently no cure for the disease. Current therapies prescribed for HD can be categorized by the symptom they treat. These categories include chorea medication, antipsychotic medication, antidepressants, mood stabilizing medication as well as non-drug therapies. Fortunately, there are also many new HD therapeutics currently undergoing clinical trials that target the disease at its origin; lowering the levels of mutant huntingtin protein (mHTT). Currently, much attention is being directed to antisense oligonucleotide (ASO) therapies, which bind to pre-RNA or mRNA and can alter protein expression via RNA degradation, blocking translation or splice modulation. Other potential therapies in clinical development include RNA interference (RNAi) therapies, RNA targeting small molecule therapies, stem cell therapies, antibody therapies, non-RNA targeting small molecule therapies and neuroinflammation targeted therapies. Potential therapies in pre-clinical development include Zinc-Finger Protein (ZFP) therapies, transcription activator-like effector nuclease (TALEN) therapies and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system (Cas) therapies. This comprehensive review aims to discuss the efficacy of current HD treatments and explore the clinical trial progress of emerging potential HD therapeutics.
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