title: Therapeutic advances in the management of Huntington's disease.
abstract: Trinucleotide repeat disorders are a set of genetic disorders characterized by the expansion of certain genes of a segment of DNA that contains a repeat of three nucleotides, thus exceeding the normal stable threshold. These repeats in the DNA cause repeats of a specific amino acid in the protein sequence, and it is the repeated amino acid that results in a defective protein. Huntington's disease is a well-known genetic disorder associated with trinucleotide repeat expansions. Patients first present clinically in midlife and manifest a typical phenotype of sporadic, rapid, and involuntary control of limb movement; stiffness of limbs; impaired cognition; severe psychiatric disturbances; and ultimately, death. There have been a number of therapeutic advances in the treatment of Huntington's disease, such as foetal neural transplantation, RNA interference, and transglutaminase inhibitor. Although there is intensive research into Huntington's disease and recent findings seem promising, effective therapeutic strategies may not be developed until the next few decades.
authors.affiliation: Cardiff University School of Medicine, School of Medicine Registry, Cardiff University, Heath Park, Cardiff CF14 4XN, United Kingdom. Abdulrahmango@cf.ac.uk
authors.forename: Ganiy Opeyemi
authors.initials: GO
authors.lastname: Abdulrahman
mesh_terms: D003538:Cystamine / Q000627:therapeutic use; D006801:Humans; D006816:Huntington Disease / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D010455:Peptides / Q000378:metabolism*; D034622:RNA Interference; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use; D057127:Single-Chain Antibodies / Q000627:therapeutic use; D013747:Tetrabenazine / Q000627:therapeutic use; D011503:Transglutaminases / Q000037:antagonists & inhibitors / Q000378:metabolism; D019680:Trinucleotide Repeat Expansion
chemical_list: D010455:Peptides; D011993:Recombinant Fusion Proteins; D057127:Single-Chain Antibodies; C097188:polyglutamine; D011503:Transglutaminases; D003538:Cystamine; D013747:Tetrabenazine