The present invention concerns compounds, compositions, and methods for
the study, diagnosis, and treatment of diseases and conditions associated
with alpha-1 antitrypsin (AAT) 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 alpha-1 antitrypsin (AAT) allelic variants
that respond to the modulation of expression and/or activity of genes
involved in alpha-1 antitrypsin (AAT) gene expression pathways or other
cellular processes that mediate the maintenance or development of alpha-1
antitrypsin (AAT) diseases and conditions such as liver disease, lung
disease, and any other diseases or conditions that are related to or will
respond to the levels of an alpha-1 antitrypsin (AAT) variant 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 (mRNA), and short hairpin RNA
(shRNA) molecules capable of mediating RNA interference (RNAi) against
the expression disease related genes or alleles having alpha-1
antitrypsin (AAT) sequences.
