Huntington's disease: An update of therapeutic strategies

Huntington's disease (HD) is an autosomal dominant triplet repeat genetic disease, which results in progressive neuronal degeneration in the neostriatum and neocortex, and associated functional impairments in motor, cognitive, and psychiatric domains. Although the genetic mutation caused by abn...

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Veröffentlicht in:Gene 2015-02, Vol.556 (2), p.91-97
Hauptverfasser: Kumar, Ashok, Kumar Singh, Sandeep, Kumar, Vijay, Kumar, Dinesh, Agarwal, Sarita, Rana, Manoj Kumar
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container_end_page 97
container_issue 2
container_start_page 91
container_title Gene
container_volume 556
creator Kumar, Ashok
Kumar Singh, Sandeep
Kumar, Vijay
Kumar, Dinesh
Agarwal, Sarita
Rana, Manoj Kumar
description Huntington's disease (HD) is an autosomal dominant triplet repeat genetic disease, which results in progressive neuronal degeneration in the neostriatum and neocortex, and associated functional impairments in motor, cognitive, and psychiatric domains. Although the genetic mutation caused by abnormal CAG expansion within the htt gene on chromosome 4p16.3 is identified, the mechanism by which this leads to neuronal cell death and the question of why striatal neurones are targeted both remain unknown. Patients manifest a typical phenotype of sporadic, rapid, involuntary control of limb movement, stiffness of limbs, impaired cognition and severe psychiatric disturbances. There have been a number of therapeutic advances in the treatment of HD, such as fetal neural transplantation, RNA interference (RNAi) and transglutaminase inhibitors (Tgasei). Although there is intensive research into HD and recent findings seem promising, effective therapeutic strategies may not be developed until the next few decades. •Genetic insight and molecular biology of the disease•Current management and therapeutics•Drugs against excitotoxicity
doi_str_mv 10.1016/j.gene.2014.11.022
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source MEDLINE; Elsevier ScienceDirect Journals
subjects Animals
Antisense oligonucleotide (ASO)
Cell- and Tissue-Based Therapy
Clinical Trials as Topic
Histone deacetylase inhibitors (HDACi)
htt gene
Humans
Huntingtin Protein
Huntington Disease - genetics
Huntington Disease - pathology
Huntington Disease - therapy
Huntington's disease (HD)
Molecular Targeted Therapy
Nerve Tissue Proteins - genetics
Nerve Tissue Proteins - metabolism
Neuroprotective Agents - pharmacology
Neuroprotective Agents - therapeutic use
Protein Aggregation, Pathological
RNA interference (RNAi)
Transglutaminase inhibitors (Tgasei)
title Huntington's disease: An update of therapeutic strategies
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