TY - JOUR
T1 - Erratum
T2 - 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))
AU - Logan, Todd
AU - Simon, Matthew J.
AU - Rana, Anil
AU - Cherf, Gerald M.
AU - Srivastava, Ankita
AU - Davis, Sonnet S.
AU - Yoon Low, Ray Lieh
AU - Chiu, Chi Lu
AU - Fang, Meng
AU - Huang, Fen
AU - Bhalla, Akhil
AU - Llapashtica, Ceyda
AU - Prorok, Rachel
AU - Pizzo, Michelle E.
AU - Calvert, Meredith E.K.
AU - Sun, Elizabeth W.
AU - Hsiao-Nakamoto, Jennifer
AU - Rajendra, Yashas
AU - Lexa, Katrina W.
AU - Srivastava, Devendra B.
AU - van Lengerich, Bettina
AU - Wang, Junhua
AU - Robles-Colmenares, Yaneth
AU - Kim, Do Jin
AU - Duque, Joseph
AU - Lenser, Melina
AU - Earr, Timothy K.
AU - Nguyen, Hoang
AU - Chau, Roni
AU - Tsogtbaatar, Buyankhishig
AU - Ravi, Ritesh
AU - Skuja, Lukas L.
AU - Solanoy, Hilda
AU - Rosen, Howard J.
AU - Boeve, Bradley F.
AU - Boxer, Adam L.
AU - Heuer, Hilary W.
AU - Dennis, Mark S.
AU - Kariolis, Mihalis S.
AU - Monroe, Kathryn M.
AU - Przybyla, Laralynne
AU - Sanchez, Pascal E.
AU - Meisner, Rene
AU - Diaz, Dolores
AU - Henne, Kirk R.
AU - Watts, Ryan J.
AU - Henry, Anastasia G.
AU - Gunasekaran, Kannan
AU - Astarita, Giuseppe
AU - Suh, Jung H.
AU - Lewcock, Joseph W.
AU - DeVos, Sarah L.
AU - Di Paolo, Gilbert
N1 - Publisher Copyright:
© 2024 Elsevier Inc.
PY - 2024/3/14
Y1 - 2024/3/14
N2 - (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.
AB - (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.
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U2 - 10.1016/j.cell.2024.02.015
DO - 10.1016/j.cell.2024.02.015
M3 - Comment/debate
C2 - 38490183
AN - SCOPUS:85187206562
SN - 0092-8674
VL - 187
SP - 1565
EP - 1566
JO - Cell
JF - Cell
IS - 6
ER -