Alpha-1-antitrypsin is a so-called protease inhibitor, a type of enzyme inhibitor. It is produced in the liver but exerts its effects in the lungs, where it regulates immune cell activity. This ...
Korro Bio Inc. has nominated its first development candidate, KRRO-110, for the potential treatment of α1-antitrypsin deficiency. KRRO-110 is a proprietary RNA editing oligonucleotide delivered to ...
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this rare ...
Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and Decreases in Mutant Z-AAT in Circulation Across ...
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