Recent Publications

High-Throughput Liquid Chromatography–Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II

International Journal of Molecular Sciences, July 30, 2020

DOI 10.3390/ijms21155449

Junhua Wang, Akhil Bhalla, Julie C. Ullman, Meng Fang, Ritesh Ravi, Annie Arguello, Elliot Thomsen, Buyankhishig Tsogtbaatar, Jing L. Guo, Lukas L. Skuja, Jason C. Dugas, Sonnet S. Davis, Suresh B. Poda, Kannan Gunasekaran, Simona Costanzo, Zachary K. Sweeney, Anastasia G. Henry, Jeffrey M. Harris, Kirk R. Henne, Giuseppe Astarita


Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients

International Journal of Molecular Sciences, July 22, 2020

DOI 10.3390/ijms21155188

Akhil Bhalla, Ritesh Ravi, Meng Fang, Annie Arguello, Sonnet S. Davis, Chi-Lu Chiu, Jessica R. Blumenfeld, Hoang N. Nguyen, Timothy K. Earr, Junhua Wang, Giuseppe Astarita, Yuda Zhu, Damian Fiore, Kimberly Scearce-Levie, Dolores Diaz, Heather Cahan, Matthew D. Troyer, Jeffrey M. Harris, Maria L. Escolar


Alzheimer’s-associated PLCγ2 is a signaling node required for both TREM2 function and the inflammatory response in human microglia

Nature, June 8, 2020

DOI 10.1038/s41593-020-0650-6

Benjamin J. Andreone, Laralynne Przybyla, Ceyda Llapashtica, Anil Rana, Sonnet S. Davis, Bettina van Lengerich, Karin Lin, Ju Shi, Yuan Mei, Giuseppe Astarita, Gilbert Di Paolo, Thomas Sandmann, Kathryn M. Monroe & Joseph W. Lewcock


Brain delivery of therapeutic proteins using a novel Fc fragment blood-brain barrier transport vehicle in mice and monkeys

Science Translational Medicine, May 27, 2020

DOI 10.1126/scitranslmed.aay1359

Mihalis S. Kariolis, Robert C. Wells, Jennifer A. Getz, Wanda Kwan, Cathal S. Mahon, Raymond Tong, Do Jin Kim, Ankita Srivastava, Catherine Bedard, Kirk R. Henne, Tina Giese, Victoria A. Assimon, Xiaocheng Chen, Yin Zhang, Hilda Solanoy, Katherine Jenkins, Pascal E. Sanchez, Lesley Kane, Takashi Miyamoto, Kylie S. Chew, Michelle E. Pizzo, Nicholas Liang, Meredith E. K. Calvert, Sarah L. DeVos, Sulochanadevi Baskaran, Sejal Hall, Zachary K. Sweeney, Robert G. Thorne, Ryan J. Watts, Mark S. Dennis, Adam P. Silverman, Y. Joy Yu Zuchero


Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice

Science Translational Medicine, May 27, 2020

DOI 10.1126/scitranslmed.aay1163

Julie C. Ullman, Annie Arguello, Jennifer A. Getz, Akhil Bhalla, Cathal S. Mahon, Junhua Wang , Tina Giese , Catherine Bedard , Do Jin Kim , Jessica R. Blumenfeld, Nicholas Liang, Ritesh Ravi, Alicia A. Nugent, Sonnet S. Davis, Connie Ha, Joseph Duque, Hai L. Tran, Robert C. Wells, Steve Lianoglou, Vinay M. Daryani, Wanda Kwan, Hilda Solanoy, Hoang Nguyen, Timothy Earr, Jason C. Dugas, Michael D. Tuck, Jennifer L. Harvey, Michelle L. Reyzer, Richard M. Caprioli, Sejal Hall, Suresh Poda, Pascal E. Sanchez, Mark S. Dennis, Kannan Gunasekaran, Ankita Srivastava, Thomas Sandmann, Kirk R. Henne, Robert G. Thorne, Gilbert Di Paolo, Giuseppe Astarita, Dolores Diaz, Adam P. Silverman, Ryan J. Watts, Zachary K. Sweeney, Mihalis S. Kariolis, Anastasia G. Henry


Leveraging preclinical models for the development of Alzheimer disease therapeutics

Nature Reviews Drug Discovery, April 8, 2020

DOI 10.1038/s41573-020-0065-9

Kimberly Scearce-Levie, Pascal E. Sanchez, Joseph W. Lewcock


Enhancing protective microglial activities with a dual function TREM 2 antibody to the stalk region

EMBO Mol Medicine, March 10, 2020

DOI 10.15252/emmm.201911227

Kai Schlepckow, Kathryn M Monroe, Gernot Kleinberger, Ludovico Cantuti‐Castelvetri, Samira Parhizkar, Dan Xia, Michael Willem, Georg Werner, Nadine Pettkus, Bettina Brunner, Alice Sülzen, Brigitte Nuscher, Heike Hampel, Xianyuan Xiang, Regina Feederle, Sabina Tahirovic, Joshua I Park, Rachel Prorok, Cathal Mahon, Chun‐Chi Liang, Ju Shi, Do Jin Kim, Hanna Sabelström, Fen Huang, Gilbert Di Paolo, Mikael Simons, Joseph W Lewcock, and Christian Haass


Review: Cell Death and Neurodegeneration

Cold Spring Harbor Perspectives, February 3, 2020

DOI 10.1101/cshperspect.a036434

Benjamin J. Andreone, Martin Larhammar, Joseph W. Lewcock


TREM2 Regulates Microglial Cholesterol Metabolism upon Chronic Phagocytic Challenge

Neuron, January 2, 2020

DOI 10.1016/j.neuron.2019.12.007

Alicia A. Nugent, Karin Lin, Bettina van Lengerich, Steve Lianoglou, Laralynne Przybyla, Sonnet S. Davis, Ceyda Llapashtica, Junhua Wang, Do Jin Kim, Dan Xia, Anthony Lucas, Sulochanadevi Baskaran, Patrick C.G. Haddick, Melina Lenser, Timothy K. Earr, Ju Shi, Jason C. Dugas, Benjamin J. Andreone, Todd Logan, Hilda O. Solanoy, Hang Chen 5, Ankita Srivastava, Suresh B. Poda, Pascal E. Sanchez, Ryan J. Watts, Thomas Sandmann, Giuseppe Astarita, Joseph W. Lewcock, Kathryn M. Monroe, Gilbert Di Paolo


DNL104, a Centrally Penetrant RIPK1 Inhibitor, Inhibits RIP 1 Kinase Phosphorylation in a Randomized Phase I Ascending Dose Study in Healthy Volunteers

Clinical Pharmacology & Therapeutics, August 22, 2019

DOI 10.1002/cpt.1615

Hendrika W. Grievink, Jules A.A.C. Heuberger, Fen Huang, Rinkal Chaudhary, Willem A.J. Birkhoff, George R. Tonn, Sofia Mosesova, Rebecca Erickson, Matthijs Moerland, Patrick C.G. Haddick, Kimberly Scearce‐Levie, Carole Ho, Geert Jan Groeneveld


Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in ALS/FTD

Neuron, July 1, 2019

DOI 10.1016/j.neuron.2019.05.048

Mark Y. Fang, Sebastian Markmiller, Anthony Q. Vu, Ashkan Javaherian, William E. Dowdle, Philippe Jolivet, Paul J. Bushway, Nicholas A. Castello, Ashmita Baral, Michelle Y. Chan, Jeremy W. Linsley, Drew Linsley, Mark Mercola, Steven Finkbeiner, Eric Lecuyer, Joseph W. Lewcock, Gene W. Yeo


SUPT4H1 Depletion Leads to a Global Reduction in RNA

Cell Reports, January 2, 2019

DOI 10.1016/j.celrep.2018.12.004

Adam Naguib, Thomas Sandmann, Fei Yi, Ryan J Watts, Joseph W Lewcock, William E Dowdle


Targeting of nonlipidated, aggregated apoE with antibodies inhibits amyloid accumulation

The Journal of Clinical Investigation, March 30, 2018

DOI 10.1172/jci96429

Fan Liao, Aimin Li, Monica Xiong, Nga Bien-Ly, Hong Jiang, Yin Zhang, Mary Beth Finn, Rosa Hoyle, Jennifer Keyser, Katheryn B Lefton, Grace O Robinson, Javier Remolina Serrano, Adam P Silverman, Jing L Guo, Jennifer Getz, Kirk Henne, Cheryl Eg Leyns, Gilbert Gallardo, Jason D Ulrich, Patrick M Sullivan, Eli Paul Lerner, Eloise Hudry, Zachary K Sweeney, Mark S Dennis, Bradley T Hyman, Ryan J Watts, David M Holtzman