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

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作者： Todd,Logan ^[1] ; Matthew J,Simon ^[1] ; Anil,Rana ^[1] ; Gerald M,Cherf ^[1] ; Ankita,Srivastava ^[1] ; Sonnet S,Davis ^[1] ; Ray Lieh Yoon,Low ^[1] ; Chi-Lu,Chiu ^[1] ; Meng,Fang ^[1] ; Fen,Huang ^[1] ; Akhil,Bhalla ^[1] ; Ceyda,Llapashtica ^[1] ; Rachel,Prorok ^[1] ; Michelle E,Pizzo ^[1] ; Meredith E K,Calvert ^[1] ; Elizabeth W,Sun ^[1] ; Jennifer,Hsiao-Nakamoto ^[1] ; Yashas,Rajendra ^[1] ; Katrina W,Lexa ^[1] ; Devendra B,Srivastava ^[1] ; Bettina,van Lengerich ^[1] ; Junhua,Wang ^[1] ; Yaneth,Robles-Colmenares ^[1] ; Do Jin,Kim ^[1] ; Joseph,Duque ^[1] ; Melina,Lenser ^[1] ; Timothy K,Earr ^[1] ; Hoang,Nguyen ^[1] ; Roni,Chau ^[1] ; Buyankhishig,Tsogtbaatar ^[1] ; Ritesh,Ravi ^[1] ; Lukas L,Skuja ^[1] ; Hilda,Solanoy ^[1] ; Howard J,Rosen ^[2] ; Bradley F,Boeve ^[3] ; Adam L,Boxer ^[2] ; Hilary W,Heuer ^[2] ; Mark S,Dennis ^[1] ; Mihalis S,Kariolis ^[1] ; Kathryn M,Monroe ^[1] ; Laralynne,Przybyla ^[1] ; Pascal E,Sanchez ^[1] ; Rene,Meisner ^[1] ; Dolores,Diaz ^[1] ; Kirk R,Henne ^[1] ; Ryan J,Watts ^[1] ; Anastasia G,Henry ^[1] ; Kannan,Gunasekaran ^[1] ; Giuseppe,Astarita ^[4] ; Jung H,Suh ^[1] ; Joseph W,Lewcock ^[1] ; Sarah L,DeVos ^[5] ; Gilbert,Di Paolo ^[6]

作者单位： Denali Therapeutics, South San Francisco, CA, USA. ^[1] Memory and Aging Center, Department of Neurology, UCSF Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, USA; On behalf of the ALLFTD investigators. ^[2] On behalf of the ALLFTD investigators; Department of Neurology, College of Medicine, Mayo Clinic, Rochester, MN, USA. ^[3] Denali Therapeutics, South San Francisco, CA, USA; Biochemistry and Molecular & Cellular Biology, Georgetown University, Washington, DC, USA. ^[4] Denali Therapeutics, South San Francisco, CA, USA. Electronic address: devos@dnli.com. ^[5] Denali Therapeutics, South San Francisco, CA, USA. Electronic address: dipaolo@dnli.com. ^[6]

关键词 GBA LBPA galectin-3 lipidomics lipids lipofuscin lysobisphosphatidic acid lysosome metabolomics neurodegenerative disease