title: Allele-selective inhibition of trinucleotide repeat genes.
abstract: Expanded trinucleotide repeats cause Huntington's disease (HD) and many other neurodegenerative disorders. There are no cures for these devastating illnesses and treatments are urgently needed. Each trinucleotide repeat disorder is the result of the mutation of just one gene, and agents that block expression of the mutant gene offer a promising option for treatment. Therapies that block expression of both mutant and wild-type alleles can have adverse effects, challenging researchers to develop strategies to lower levels of mutant protein while leaving adequate wild-type protein levels. Here, we review approaches that use synthetic nucleic acids to inhibit expression of trinucleotide repeat genes.
authors.affiliation: Departments of Pharmacology and Biochemistry, University of Texas Southwestern Medical Center, 6001 Forest Park Road, Dallas, TX 75390-9041, USA.
authors.forename: Masayuki
authors.initials: M
authors.lastname: Matsui
authors.forename: David R
authors.initials: DR
authors.lastname: Corey
mesh_terms: D000483:Alleles; D000818:Animals; D020868:Gene Silencing; D006801:Humans; D019636:Neurodegenerative Diseases / Q000188:drug therapy* / Q000235:genetics; D009696:Nucleic Acids / Q000494:pharmacology / Q000627:therapeutic use*; D011110:Polymorphism, Genetic; D019680:Trinucleotide Repeat Expansion / Q000235:genetics*
chemical_list: D009696:Nucleic Acids