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., Low, Ray Lieh Yoon, 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., Lewcock, Joseph W., DeVos, Sarah L., Di Paolo, Gilbert

Source: In Cell 2 September 2021 184(18):4651-4668

DNL343 is an investigational CNS penetrant eukaryotic initiation factor 2B activator that prevents and reverses the effects of neurodegeneration caused by the integrated stress response.

Authors: Yulyaningsih, Ernie, Suh, Jung H., Fanok, Melania, Chau, Roni, Solanoy, Hilda, Takahashi, Ryan, Bakardjiev, Anna I., Becerra, Isabel, Benitez, N. Butch, Chi-Lu Chiu, Davis, Sonnet S., Dowdle, William E., Earr, Timothy, Estrada, Anthony A., Gill, Audrey, Ha, Connie, Haddick, Patrick C. G., Henne, Kirk R., Larhammar, Martin, Leung, Amy W-S.

Source: eLife; 9/17/2024, p1-32, 32p

Fibrillar Aβ causes profound microglial metabolic perturbations in a novel APP knock‐in mouse model

Authors: Xia, Dan, Lianoglou, Steve, Sandmann, Thomas, Calvert, Meredith, Suh, Jung, Thomsen, Elliot, Dugas, Jason, Pizzo, Michelle E., DeVos, Sarah L., Earr, Timothy K., Lin, Chia‐Ching, Davis, Sonnet, Ha, Connie, Nguyen, Hoang, Chau, Roni, Yulyaningsih, Ernie, Solanoy, Hilda, Masoud, Shababa T., Liang, Richard, Lin, Karin, Thorne, Robert G., Garceau, Dylan, Whitesell, Jennifer D, Sasner, Michael, Harris, Julie A, Scearce‐Levie, Kimberly, Lewcock, Joseph W., Paolo, Gilbert Di, Sanchez, Pascal E.

Source: Alzheimer's & dementia. 17(3)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100139016776.0x000001&indx=1&recIds=ETOCvdc_100139016776.0x000001

Molecular architecture determines brain delivery of a transferrin receptor—targeted lysosomal enzyme

Authors: Arguello, Annie, Mahon, Cathal S., Calvert, Meredith E. K., Chan, Darren, Dugas, Jason C., Pizzo, Michelle E., Thomsen, Elliot R., Chau, Roni, Damo, Lorna A., Duque, Joseph

Source: Journal of experimental medicine. 219(3):E20211057-E20211057

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN628810537&indx=1&recIds=ETOCRN628810537

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., Lewcock, Joseph W., DeVos, Sarah L., Di Paolo, Gilbert

Source: In Cell 14 March 2024 187(6):1565-1566

Discovery of Potent and Selective Dual Leucine Zipper Kinase/Leucine Zipper-Bearing Kinase Inhibitors with Neuroprotective Properties in In Vitro and In Vivo Models of Amyotrophic Lateral Sclerosis.

Authors: Craig II, Robert A., Fox, Brian M., Hu, Cheng, Lexa, Katrina W., Osipov, Maksim, Thottumkara, Arun P., Larhammar, Martin, Miyamoto, Takashi, Rana, Anil, Kane, Lesley A., Yulyaningsih, Ernie, Solanoy, Hilda, Nguyen, Hoang, Chau, Roni, Earr, Timothy, Kajiwara, Yuji, Fleck, Daniel, Lucas, Anthony, Haddick, Patrick C. G., Takahashi, Ryan H.

Source: Journal of Medicinal Chemistry; 12/22/2022, Vol. 65 Issue 24, p16290-16312, 23p

Novel App knock-in mouse model shows key features of amyloid pathology and reveals profound metabolic dysregulation of microglia.

Authors: Xia, Dan, Lianoglou, Steve, Sandmann, Thomas, Calvert, Meredith, Suh, Jung H., Thomsen, Elliot, Dugas, Jason, Pizzo, Michelle E., DeVos, Sarah L., Earr, Timothy K., Lin, Chia-Ching, Davis, Sonnet, Ha, Connie, Leung, Amy Wing-Sze, Nguyen, Hoang, Chau, Roni, Yulyaningsih, Ernie, Lopez, Isabel, Solanoy, Hilda, Masoud, Shababa T.

Source: Molecular Neurodegeneration; 6/11/2022, Vol. 17 Issue 1, p1-29, 29p

Subject Terms: AMYLOID plaque, MICROGLIA, LABORATORY mice, ANIMAL disease models, PATHOLOGY, AMYLOID, ALZHEIMER'S disease

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