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Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.

作者信息

Logan Todd, Simon Matthew J, Rana Anil, Cherf Gerald M, Srivastava Ankita, Davis Sonnet S, Yoon Low Ray Lieh, Chiu Chi-Lu, Fang Meng, Huang Fen, Bhalla Akhil, Llapashtica Ceyda, Prorok Rachel, Pizzo Michelle E, Calvert Meredith E K, Sun Elizabeth W, Hsiao-Nakamoto Jennifer, Rajendra Yashas, Lexa Katrina W, Srivastava Devendra B, van Lengerich Bettina, Wang Junhua, Robles-Colmenares Yaneth, Kim Do Jin, Duque Joseph, Lenser Melina, Earr Timothy K, Nguyen Hoang, Chau Roni, Tsogtbaatar Buyankhishig, Ravi Ritesh, Skuja Lukas L, Solanoy Hilda, Rosen Howard J, Boeve Bradley F, Boxer Adam L, Heuer Hilary W, Dennis Mark S, Kariolis Mihalis S, Monroe Kathryn M, Przybyla Laralynne, Sanchez Pascal E, Meisner Rene, Diaz Dolores, Henne Kirk R, Watts Ryan J, Henry Anastasia G, Gunasekaran Kannan, Astarita Giuseppe, Suh Jung H, Lewcock Joseph W, DeVos Sarah L, Di Paolo Gilbert

出版信息

Cell. 2024 Mar 14;187(6):1565-1566. doi: 10.1016/j.cell.2024.02.015.

DOI:10.1016/j.cell.2024.02.015
PMID:38490183
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11089899/
Abstract
摘要

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