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

Title: Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic
Authors: 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.
Source: Cell ; volume 187, issue 6, page 1565-1566 ; ISSN 0092-8674
Publisher Information: Elsevier BV
Publication Year: 2024
Collection: ScienceDirect (Elsevier - Open Access Articles via Crossref)
Document Type: article in journal/newspaper
Language: English
DOI: 10.1016/j.cell.2024.02.015
Availability: https://doi.org/10.1016/j.cell.2024.02.015; https://api.elsevier.com/content/article/PII:S0092867424001818?httpAccept=text/xml; https://api.elsevier.com/content/article/PII:S0092867424001818?httpAccept=text/plain
Rights: https://www.elsevier.com/tdm/userlicense/1.0/ ; https://www.elsevier.com/legal/tdmrep-license ; http://www.elsevier.com/open-access/userlicense/1.0/ ; https://doi.org/10.15223/policy-017 ; https://doi.org/10.15223/policy-037 ; https://doi.org/10.15223/policy-012 ; https://doi.org/10.15223/policy-029 ; https://doi.org/10.15223/policy-004
Accession Number: edsbas.1092E798
Database: BASE