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While current therapies and those in development may ameliorate lung or liver disease, few are designed to address both. We have developed SERPINA1-994, an N-Acetylgalactosamine-conjugated chemically modified oligonucleotide that elicits adenine-to-inosine RNA editing using endogenous adenosine deaminases acting on RNA enzymes, to correct\u00a0SERPINA1 Z transcripts. We show that SERPINA1-994 edits 50% of the Z transcript in hepatocytes of NSG-PiZ mice, which increases total serum AAT levels and induces the production of wild-type M-AAT protein. SERPINA1-994 addresses loss of AAT function in lung by increasing the neutrophil elastase inhibition capacity of mouse serum and gain of function in liver by correcting gene expression patterns, decreasing Z-AAT protein aggregation and decreasing inflammation. SERPINA1-994 relies on a clinically proven delivery technology and directs highly specific RNA rather than DNA editing with no bystander editing. Overall, these data suggest that SERPINA1-994 changes a ZZ homozygous state, which is associated with a high risk for AATD, to a low-risk MZ-like phenotype.<\/jats:p>","DOI":"10.1093\/nar\/gkaf1518","type":"journal-article","created":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T12:49:12Z","timestamp":1767271752000},"source":"Crossref","is-referenced-by-count":4,"title":["RNA editing for the treatment of alpha-1 antitrypsin deficiency"],"prefix":"10.1093","volume":"54","author":[{"given":"Prashant","family":"Monian","sequence":"first","affiliation":[{"name":"Wave Life Sciences , Cambridge, MA 02138 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chikdu","family":"Shivalila","sequence":"additional","affiliation":[{"name":"Wave Life Sciences , Cambridge, MA 02138 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Genliang","family":"Lu","sequence":"additional","affiliation":[{"name":"Wave Life 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