Therapeutic Advances for Huntington’s Disease.

Publication date: Jan 12, 2020

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.

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Kumar, A., , Kumar, Singh, K., Kumar, S., Kim, Y.S., Lee, Y.M., and Kim, J.J. Therapeutic Advances for Huntington’s Disease. 06887. 2020 Brain Sci (10):1.

Concepts Keywords
Adenine Chorea
Autosomal HTT
Caspases HD
Chorea Neurodegeneration
Chromosome Nervous system
Cognitive Impairment Huntingtin
Cytosine Huntington’s disease
Depression Organ systems
Dopamine Pathways Branches of biology
Fetal Motor cognitive impairment
Gene Neurological disorders
Impaired Cognition
Neurological Disorders


Type Source Name
disease MESH muscular atrophy
drug DRUGBANK L-Tryptophan
drug DRUGBANK Vorinostat
drug DRUGBANK Icosapent
drug DRUGBANK Proline
drug DRUGBANK Phenylbutyric acid
drug DRUGBANK Trehalose
drug DRUGBANK Sirolimus
pathway KEGG Autophagy
disease MESH atrophy
drug DRUGBANK Tilmicosin
drug DRUGBANK Minocycline
drug DRUGBANK Tetracycline
drug DRUGBANK Risperidone
disease MESH dementia
drug DRUGBANK Ketamine
disease MESH amyotrophic lateral sclerosis
drug DRUGBANK Memantine
drug DRUGBANK Riluzole
drug DRUGBANK Nitric Oxide
disease MESH death
pathway KEGG Apoptosis
disease MESH oxidative stress
drug DRUGBANK Amino acids
disease MESH tryptophan
drug DRUGBANK Laquinimod
drug DRUGBANK Tauroursodeoxycholic acid
drug DRUGBANK Amantadine
drug DRUGBANK Latrepirdine
drug DRUGBANK Triheptanoin
disease MESH defects
drug DRUGBANK Pridopidine
disease MESH shock
drug DRUGBANK Glutathione
drug DRUGBANK Cysteamine
drug DRUGBANK Uric Acid
drug DRUGBANK Tiapride
disease MESH complications
disease MESH peripheral neuropathy
drug DRUGBANK Alpha-methyltryptamine
disease MESH spinal muscular atrophy
disease MESH thrombocytopenia
disease MESH stop codon
drug DRUGBANK Plicamycin
drug DRUGBANK Meclizine
drug DRUGBANK Ubidecarenone
drug DRUGBANK Creatine
drug DRUGBANK Zinc
drug DRUGBANK Ammonia
drug DRUGBANK L-Glutamine
disease MESH Huntington chorea
drug DRUGBANK Deutetrabenazine
drug DRUGBANK Tetrabenazine
disease MESH itching
disease MESH tics
drug DRUGBANK Ethanol
disease MESH depression
disease MESH chorea
disease MESH involuntary movement
disease MESH pathology
drug DRUGBANK Adenine
drug DRUGBANK Guanine
disease MESH cognitive impairment
disease MESH incoordination
disease MESH psychosis
disease MESH neurodegenerative disease
drug DRUGBANK Coenzyme M
drug DRUGBANK Ademetionine
disease MESH neurological disorders
drug DRUGBANK Dopamine
pathway KEGG Huntington disease
disease MESH Risk factors
disease MESH Substance abuse
disease MESH growth
disease MESH diagnoses
drug DRUGBANK Isoxaflutole
drug DRUGBANK Guanosine
drug DRUGBANK Atorvastatin
disease MESH multiple
disease MESH repression
drug DRUGBANK Remacemide
disease MESH Motor neuron disease
drug DRUGBANK Ferrous sulfate anhydrous
disease MESH intermittent fasting
drug DRUGBANK Lamotrigine
disease MESH bipolar disorder
drug DRUGBANK Selisistat


Original Article

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