title: HD Therapeutics - CHDI Fifth Annual Conference.
abstract: The CHDI Fifth Annual HD Therapeutics Conference, held in Palm Springs, CA, included topics covering new therapeutic developments in the field of Huntington's disease (HD). This conference report highlights presentations on biomarkers in HD; emerging topics in drug targeting, such as the lysosomal degradation pathway and target prediction by network-based modeling; understanding phenotype and neuronal circuit dysfunction in animal models; regulation of huntingtin protein expression and function; RNAi and antisense technology to deplete the mutant huntingtin protein; and small-molecule drugs that are progressing quickly through the clinic. Investigational drugs discussed include ALN-HTT (Alnylam Pharmaceuticals Inc/Medtronic Inc), EPI-743 (Edison Pharmaceuticals Inc), LNK-754 (Link Medicine Corp) and pridopidine (NeuroSearch A/S).
authors.affiliation: University of Texas, Southwestern Medical Center, Departments of Pharmacology and Biochemistry, Dallas, TX 75390-9041, USA. keith.gagnon@utsouthwestern.edu
authors.forename: Keith T
authors.initials: KT
authors.lastname: Gagnon
mesh_terms: D000818:Animals; D015415:Biomarkers / Q000378:metabolism; D004195:Disease Models, Animal; D004298:Dopamine / Q000378:metabolism; D016503:Drug Delivery Systems / Q000379:methods; D004791:Enzyme Inhibitors / Q000494:pharmacology / Q000627:therapeutic use; D051231:Farnesyltranstransferase / Q000037:antagonists & inhibitors; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000175:diagnosis / Q000378:metabolism / Q000503:physiopathology / Q000628:therapy*; D007888:Leigh Disease / Q000188:drug therapy / Q000235:genetics; D008247:Lysosomes / Q000378:metabolism; D008954:Models, Biological; D009419:Nerve Tissue Proteins / Q000235:genetics / Q000378:metabolism; D019636:Neurodegenerative Diseases / Q000188:drug therapy; D009687:Nuclear Proteins / Q000235:genetics / Q000378:metabolism; D016376:Oligonucleotides, Antisense / Q000235:genetics / Q000627:therapeutic use; D010766:Phosphorylation / Q000187:drug effects; D010880:Piperidines / Q000627:therapeutic use; D034741:RNA, Small Interfering / Q000235:genetics / Q000627:therapeutic use; D012756:Sheep; D056564:Sirtuin 1 / Q000235:genetics / Q000378:metabolism; D014451:Ubiquinone / Q000031:analogs & derivatives / Q000627:therapeutic use
chemical_list: D015415:Biomarkers; D004791:Enzyme Inhibitors; C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D016376:Oligonucleotides, Antisense; D010880:Piperidines; D034741:RNA, Small Interfering; D014451:Ubiquinone; D051231:Farnesyltranstransferase; C447939:SIRT1 protein, human; D056564:Sirtuin 1; C483720:pridopidine; D004298:Dopamine