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Therapeutic Advances for Huntington’s Disease

Department of Genetics, Sanjay Gandhi Post-Graduate Institute of Medical Sciences, Lucknow 226014, UP, India
Department of Biotechnology, Yeungnam University, Gyeongsan, Gyeongbuk 38541, Korea
Central Research Station, Subharti Medical College, Swami Vivekanand Subharti University, Meerut 250002, India
PG Department of Botany, Nalanda College, Bihar Sharif, Magadh University, Bihar 824234, India
Authors to whom correspondence should be addressed.
Contributed equally as co-first authors.
Brain Sci. 2020, 10(1), 43;
Received: 5 November 2019 / Revised: 9 January 2020 / Accepted: 10 January 2020 / Published: 12 January 2020
(This article belongs to the Special Issue Juvenile Onset Huntington's Disease)
Huntington’s disease (HD) is a progressive neurological disease that is inherited in an autosomal fashion. The cause of disease pathology is an expansion of cytosine-adenine-guanine (CAG) repeats within the huntingtin gene (HTT) on chromosome 4 (4p16.3), which codes the huntingtin protein (mHTT). The common symptoms of HD include motor and cognitive impairment of psychiatric functions. Patients exhibit a representative phenotype of involuntary movement (chorea) of limbs, impaired cognition, and severe psychiatric disturbances (mood swings, depression, and personality changes). A variety of symptomatic treatments (which target glutamate and dopamine pathways, caspases, inhibition of aggregation, mitochondrial dysfunction, transcriptional dysregulation, and fetal neural transplants, etc.) are available and some are in the pipeline. Advancement in novel therapeutic approaches include targeting the mutant huntingtin (mHTT) protein and the HTT gene. New gene editing techniques will reduce the CAG repeats. More appropriate and readily tractable treatment goals, coupled with advances in analytical tools will help to assess the clinical outcomes of HD treatments. This will not only improve the quality of life and life span of HD patients, but it will also provide a beneficial role in other inherited and neurological disorders. In this review, we aim to discuss current therapeutic research approaches and their possible uses for HD. View Full-Text
Keywords: Huntington’s disease; CAG repeat; mutant huntingtin (mHTT); therapeutics; neurodegeneration Huntington’s disease; CAG repeat; mutant huntingtin (mHTT); therapeutics; neurodegeneration
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MDPI and ACS Style

Kumar, A.; Kumar, V.; Singh, K.; Kumar, S.; Kim, Y.-S.; Lee, Y.-M.; Kim, J.-J. Therapeutic Advances for Huntington’s Disease. Brain Sci. 2020, 10, 43.

AMA Style

Kumar A, Kumar V, Singh K, Kumar S, Kim Y-S, Lee Y-M, Kim J-J. Therapeutic Advances for Huntington’s Disease. Brain Sciences. 2020; 10(1):43.

Chicago/Turabian Style

Kumar, Ashok; Kumar, Vijay; Singh, Kritanjali; Kumar, Sukesh; Kim, You-Sam; Lee, Yun-Mi; Kim, Jong-Joo. 2020. "Therapeutic Advances for Huntington’s Disease" Brain Sci. 10, no. 1: 43.

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