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BioWorld - Saturday, November 8, 2025
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» Preclinical data on single-dose gene editing system for AATD
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Endocrine/metabolic
Preclinical data on single-dose gene editing system for AATD
Oct. 21, 2025
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Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the
SERPINA1
gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
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