Allele-selective inhibition of trinucleotide repeat genes

M Matsui, DR Corey - Drug discovery today, 2012 - Elsevier
M Matsui, DR Corey
Drug discovery today, 2012Elsevier
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 …
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.
Elsevier