Article
Dual SORT LNPs for multi-organ base editing.
Nature biotechnology - 1 Apr 2026
Kim Minjeong, Song Eunice S, Chen Joseph C, Chatterjee Sumanta, Sun Yehui, Lee Sang M, Wu Shiying, Patel Priyanka, Tian Zeru, Kantor Ariel, Wustman Brandon A, Lockhart David J, Siegwart Daniel J
Abstract excerpt
Alpha-1 antitrypsin (A1AT) deficiency (AATD) is caused by a mutation in the SERPINA1 gene (PiZ allele), where misfolded A1AT liver accumulation leads to liver damage, and A1AT deficiency in the lungs results in emphysema due to unregulated neutrophil elastase activity. Base editing offers a potential cure for A1AT; however, effective treatment is hindered by the absence of dual-target delivery systems that can...
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