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 |
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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 |
format | Article |
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Kumar Singh, Sandeep ; Kumar, Vijay ; Kumar, Dinesh ; Agarwal, Sarita ; Rana, Manoj Kumar</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c415t-66410a2d1ef544a990317f7363c1c7257163269543c620bcefabd9a89a6c1b763</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2015</creationdate><topic>Animals</topic><topic>Antisense oligonucleotide (ASO)</topic><topic>Cell- and Tissue-Based Therapy</topic><topic>Clinical Trials as Topic</topic><topic>Histone deacetylase inhibitors (HDACi)</topic><topic>htt gene</topic><topic>Humans</topic><topic>Huntingtin Protein</topic><topic>Huntington Disease - genetics</topic><topic>Huntington Disease - pathology</topic><topic>Huntington Disease - therapy</topic><topic>Huntington's disease (HD)</topic><topic>Molecular Targeted Therapy</topic><topic>Nerve Tissue Proteins - genetics</topic><topic>Nerve Tissue Proteins - metabolism</topic><topic>Neuroprotective Agents - pharmacology</topic><topic>Neuroprotective Agents - therapeutic use</topic><topic>Protein Aggregation, Pathological</topic><topic>RNA interference (RNAi)</topic><topic>Transglutaminase inhibitors (Tgasei)</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Kumar, Ashok</creatorcontrib><creatorcontrib>Kumar Singh, Sandeep</creatorcontrib><creatorcontrib>Kumar, Vijay</creatorcontrib><creatorcontrib>Kumar, Dinesh</creatorcontrib><creatorcontrib>Agarwal, Sarita</creatorcontrib><creatorcontrib>Rana, Manoj Kumar</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Neurosciences Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Gene</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Kumar, Ashok</au><au>Kumar Singh, Sandeep</au><au>Kumar, Vijay</au><au>Kumar, Dinesh</au><au>Agarwal, Sarita</au><au>Rana, Manoj Kumar</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Huntington's disease: An update of therapeutic strategies</atitle><jtitle>Gene</jtitle><addtitle>Gene</addtitle><date>2015-02-10</date><risdate>2015</risdate><volume>556</volume><issue>2</issue><spage>91</spage><epage>97</epage><pages>91-97</pages><issn>0378-1119</issn><eissn>1879-0038</eissn><abstract>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.
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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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