title: Huntington's disease: an update of therapeutic strategies.
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.
authors.affiliation: Department of Genetics, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow 226014, India. Electronic address: chemistry.ashok83@gmail.com.
authors.forename: Ashok
authors.initials: A
authors.lastname: Kumar
authors.affiliation: Department of Neurology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow 226014, India. Electronic address: sandeepcbt@gmail.com.
authors.forename: Sandeep
authors.initials: S
authors.lastname: Kumar Singh
authors.affiliation: Department of Neurology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow 226014, India. Electronic address: vijaykumarcbt@gmail.com.
authors.forename: Vijay
authors.initials: V
authors.lastname: Kumar
authors.affiliation: Department of Chemistry, Dr. Ram Manohar Lohia Avadh University, Faizabad 224001, India. Electronic address: masterdinesh87@gmail.com.
authors.forename: Dinesh
authors.initials: D
authors.lastname: Kumar
authors.affiliation: Department of Genetics, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow 226014, India. Electronic address: saritasgpgi@gmail.com.
authors.forename: Sarita
authors.initials: S
authors.lastname: Agarwal
authors.affiliation: Department of Microbiology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow-226014, India. Electronic address: manojkumarranapgi@gmail.com.
authors.forename: Manoj Kumar
authors.initials: MK
authors.lastname: Rana
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy; D002986:Clinical Trials as Topic; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000235:genetics / Q000473:pathology* / Q000628:therapy*; D058990:Molecular Targeted Therapy; D009419:Nerve Tissue Proteins / Q000235:genetics* / Q000378:metabolism; D018696:Neuroprotective Agents / Q000494:pharmacology / Q000627:therapeutic use; D066263:Protein Aggregation, Pathological
chemical_list: C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D018696:Neuroprotective Agents
keywords: Antisense oligonucleotide (ASO); Histone deacetylase inhibitors (HDACi); Huntington's disease (HD); RNA interference (RNAi); Transglutaminase inhibitors (Tgasei); htt gene