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

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

2021Cell229 citationsDOIOpen Access PDF

Topics & Concepts

BiologyFrontotemporal dementiaPenetrant (biochemical)Loss functionFunction (biology)Frontotemporal lobar degenerationBrain functionNeuroscienceNeurodegenerationCell biologyGeneticsPhenotypeDementiaInternal medicineGeneDiseaseBiotechnologyMedicineAmyotrophic Lateral Sclerosis ResearchAlzheimer's disease research and treatmentsParkinson's Disease Mechanisms and Treatments
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic | Litcius