Erratum: Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic (Cell (2021) 184(18) (4651–4668.e25), (S0092867421009442), (10.1016/j.cell.2021.08.002))

Todd Logan, Matthew J. Simon, Anil Rana, Gerald M. Cherf, Ankita Srivastava, Sonnet S. Davis, Ray Lieh Yoon Low, Chi Lu Chiu, Meng Fang, Fen Huang, Akhil Bhalla, Ceyda Llapashtica, Rachel Prorok, Michelle E. Pizzo, Meredith E.K. Calvert, Elizabeth W. Sun, Jennifer Hsiao-Nakamoto, Yashas Rajendra, Katrina W. Lexa, Devendra B. SrivastavaBettina van Lengerich, Junhua Wang, Yaneth Robles-Colmenares, Do Jin Kim, Joseph Duque, Melina Lenser, Timothy K. Earr, Hoang 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, Rene 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

Research output: Contribution to journalComment/debatepeer-review

Abstract

(Cell 184, 4651–4668.e1–e25; September 2, 2021) In the originally published version of this article, we reported tissue and fluid concentrations of PTV:PGRN and Fc:PGRN following IV administration in a subset of panels from Figures 5 and S5. Following publication, further characterization of PTV:PGRN and assay optimization for drug pharmacokinetic profiling was conducted as part of the ongoing clinical investigation of PTV:PGRN. During these efforts, we discovered that the tissue drug concentrations reported in this manuscript did not account for a uniform, 10-fold dilution factor introduced during tissue homogenization. As a result, the y-axis values in Figures 5C, S5B, and S5D should be one order of magnitude higher than originally reported. To validate this finding, the study was repeated with the original assay format and drug doses, and the dilution factor error was confirmed. This calculation error does not alter the relative relationship of drug uptake in the brain between doses and molecule architecture, nor does it impact the conclusions drawn based on the original data, but it does suggest that brain uptake of drug is 10-fold higher than previously reported. No changes were made in the text of the manuscript, only in the figures. The corrected Figures 5C, S5B, and S5D are shown here. We apologize for any confusion this error may have caused.

Original languageEnglish (US)
Pages (from-to)1565-1566
Number of pages2
JournalCell
Volume187
Issue number6
DOIs
StatePublished - Mar 14 2024

ASJC Scopus subject areas

  • General Biochemistry, Genetics and Molecular Biology

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