Intellia Therapeutics, Inc.
Compositions and Methods for Treating Alpha-1 Antitrypsin Deficiency

Abstract:

Compositions and methods for introducing double-stranded breaks within the SERPINA1 gene are provided. Compositions and methods for reducing and eliminating mutant forms of al-antitrypsin (AAT), such as seen in subjects having al-antitrypsin deficiency (AATD), are provided.