The present invention concerns compounds, compositions, and methods for
the study, diagnosis, and treatment of diseases and conditions associated
with polyglutamine repeat (polyQ) allelic variants that respond to the
modulation of gene expression and/or activity. The present invention also
concerns compounds, compositions, and methods relating to diseases and
conditions associated with polyglutamine repeat (polyQ) allelic variants
that respond to the modulation of expression and/or activity of genes
involved in polyQ repeat gene expression pathways or other cellular
processes that mediate the maintenance or development of polyQ repeat
diseases and conditions such as Huntinton disease and related conditions
such as progressive chorea, rigidity, dementia, and seizures,
spinocerebellar ataxia, spinal and bulbar muscular dystrophy (SBMA),
dentatorubropallidoluysian atrophy (DRPLA), and any other diseases or
conditions that are related to or will respond to the levels of a repeat
expansion (RE) protein in a cell or tissue, alone or in combination with
other therapies. Specifically, the invention relates to small nucleic
acid molecules, such as short interfering nucleic acid (siNA), short
interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA),
and short hairpin RNA (shRNA) molecules capable of mediating RNA
interference (RNAi) against the expression disease related genes or
alleles having polyQ repeat sequences.
