| 摘要 |
<p>The present invention relates to methods and compositions for modulating the unfolded protein response. The method further relates to methods and compositions for the treatment and diagnosis of protein conformational diseases or disorders, including, but not limited to, α1-antitrypsin deficiency, cystic fibrosis, and autoimmune diseases and disorders. The invention further provides methods for modulating the unfolded protein response by modulating XBP1 mRNA splicing.</p> |
