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Huntington’s Disease: Mechanisms of Pathogenesis and Therapeutic Strategies
Open accessAbstract
Huntington's disease is a late-onset neurodegenerative disease caused by a CAG trinucleotide repeat in the gene encoding the huntingtin protein. Despite its well-defined genetic origin, the molecular and cellular mechanisms underlying the disease are unclear and complex. Here, we review some of the currently known functions of the wild-type huntingtin protein and discuss the deleterious effects that arise from the expansion of the CAG repeats, which are translated into an abnormally long polyglutamine tract. Finally, we outline some of the therapeutic strategies that are currently being pursued to slow down the disease.
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Metadata
- Authors
- Jimenez-Sanchez, M., Licitra, F., Underwood, B. R., & Rubinsztein, D. C.
- Venue
- Cold Spring Harbor Perspectives in Medicine
- Publisher
- Cold Spring Harbor Laboratory
- Published
- 2016-12-09
- Volume
- 7
- Issue
- 7
- Pages
- a024240
- ISSN
- 2157-1422 (ISSN-L)
- OpenAlex
- W2559796913
- Cited by
- 507
- References
- 253
- Type
- posted content
- Language
- en
- Open access
- bronze
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