LNP-AAV Hybrid Delivery of a DNA Gene Writer System Corrects Severe Ornithine Transcarbamylase Deficiency in Neonatal Mice and Improves AAV Potency in Adult Mouse and NHP Livers

Philip Zakas,Sharon C. Cunningham, Ann Doherty, Stephanie Yu, Bef Mekonnen,Raed Ibraheim, Brendan Lang,Elizabeth English, John Solitro, Mayuri Singh, Eva B. van Dijk, Gang Sun, Miles Duncan, Matthew Benczkowski, Robert C. Altshuler, Malvenderjit Jagjit Singh, Emily Kibbler,Gulen Tonga, Jane Wang, Guangde Li, Jeff An,James B. Rottman, Yanfang Fu, Robert J. Citorik, Jacob R. Rubens,Cecilia Cotta-Ramusino,William Querbes,Ian E. Alexander, William E. Salomon

MOLECULAR THERAPY(2023)

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