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Faculty

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Mariko Bennett, MD, PhD

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Assistant Professor of Neurology
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Department: Neurology
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1f Graduate Group Affiliations 8 a
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46 Contact information
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3501 Civic Center Blvd
2e Colket Translational Research Building
36 10th floor 10-10300
Philadelphia, PA 19104
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43 Lab: 267-425-9566
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18 Publications
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13 Education:
21 7 BS 35 (Behavioral Neuroscience, Summa Cum Laude) c
30 Northeastern University, 2007.
21 b MD/PhD 19 (Neuroscience) c
63 Medical Scientist Training Program, Stanford University School of Medicine, 2018.
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Description of Clinical Expertise

98 I am a pediatric neurologist who mostly sees patients in the Leukodystrophy Center for Excellence at Children's Hospital of Philadelphia
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Description of Research Expertise

14c We study how microglia – the brain’s tissue-resident macrophages—contribute to development and disease, and how this knowledge can inform therapies. As a child neurologist, I care for children with genetic and acquired neuroimmunological disorders including leukodystrophies and genetic leukoencephalopathies.
30d Over the past decade, I’ve created a comprehensive platform to study microglia that includes identification, culture, sequencing, transplantation, and in vivo manipulation of these cells. My vision is to use these techniques to transform neurological disease treatment by creating microglia-based cell and gene therapies. Based on my scientific background and clinical experience, I have more recently become interested in how microglia protect the brain from viral infection, how these pathways go awry in genetic inflammatory diseases, and how to bridge basic science with the clinic bringing the tools of next-generation sequencing together with mouse xenotransplant and human organoid models to unlock new therapeutic avenues for pediatric neuroinflammatory disorders.
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Selected Publications

b1 Bennett ML, Bennett FC.: CNS engraftment after HCT: Making the niche more accessible. Mol Ther May 2026.

152 Gonzalez Saez-Diez E, Ferrer Socorro M, Yang K, Battaglia N, Zaman Z, Bennett M, Vanderver A, Lee PY, Henderson LA, Andzelm MM, Ebrahimi-Fakhari D.: Movement Disorders in Aicardi-Goutières Syndrome and Response to Immunomodulation. Ann Clin Transl Neurol Apr 2026.

305 Calame DG, Wiener EK, Gavazzi F, Sevagamoorthy A, Pizzino A, Arnold K, Dominguez Gonzalez C, Jammihal T, Bennett M, Adang L, Woidill S, Whitehead MT, Vossough A, D'Aiello R, Takanohashi A, Lele J, Simons C, Rius R, Formaini E, Sullivan KE, Andzelm M, Ebrahimi-Fakhari D, Otten C, Wong S, Reynolds T, Schiffmann R, Wolf NI, Waisfisz Q, Niermeijer JM, DeMarzo D, Dawood M, Gandhi M, Levine JM, Chinn IK, Fisher K, Emrick L, Alam CA, Kaiyrzhanov R, Maroofian R, Houlden H, Jhangiani SN, Mehta HH, Muzny DM, Sedlazeck FJ, Posey JE, Lupski JR, Gibbs RA, Rajagopalan R, Vanderver A.: PTPN1-related autoinflammation is a common cause of Aicardi-Goutières Syndrome with reduced penetrance. medRxiv Apr 2026.

1e3 Nemec KM, Uy G, Chaluvadi VS, Purnell FS, Elfayoumi B, Byerly L, O'Reilly ML, O'Brien CA, Aisenberg WH, Lombroso SI, Guo X, Blank N, Oon CH, Yaqoob F, Temsamrit B, Rawat P, Thaiss CA, Bailis W, Williamson AP, Wang Q, Bennett ML, Bennett FC.: Microglia replacement by ER-Hoxb8 conditionally immortalized macrophages provides insight into Aicardi-Goutières syndrome neuropathology. Elife 14: RP102900, Jan 2026.

e8 McKinney JE, Bennett ML, Bennett FC.: Basic Microglial Functions and How They Go Awry in Neurodegenerative Disease. Annu Rev Pathol 21(1): 59-80, January 2026.

151 Carleigh A. O’Brien, Samuelle A.S. Delcy, Sangeeta Shukla, Eli M. Levitt, Akiva S. Cohen, Mariko L. Bennett, Frederick C. Bennett: Neonatal microglia replacement in mice modulates seizure severity in adulthood. Molecular Therapy 33(12): 6248-6264, December 2025.

2ea Sonia I Lombroso, Emily I Church, Ryan N Anderson, Carleigh A O’Brien, Yixuan Zhu, Asif A Dar, Freddy S Purnell, Eli M Levitt, Brian Temsamrit, Fazeela Yaqoob, William H Aisenberg, Mara Davis, Bilal Elfayoumi, Sai Chaluvadi, Kelsey M Nemec, Jessie Axsom, Daniel A Shapiro, Chet Huan Oon, Jean Paul Chadarevian, Charles-Antoine Assenmacher, Stefano De Arcangeli, Esha Banerjee, Sangeeta Shukla, Hayk Davtyan, Will Bailis, Christopher A Hunter, Mathew Blurton-Jones, Mariko L Bennett, Michael S Haney, F Chris Bennett: Microglia replacement by peripheral delivery of CSF1R inhibitor-resistant hematopoietic cells. Molecular Therapy 34: S1525-0016(25)00871-8, November 2025.

108 Ryan J Rahman, Mariko L Bennett: Microglial MEF2C is a uniquely human bridge between neurodevelopment and neurodegeneration. Nature Immunology 26(11): 1847-1848, November 2025 Notes: Preview.

279 Nicolas Marzolini, Taylor V Brysgel, Ryan J Rahman, Eno-Obong Essien, Saw Y Nwe, Jichuan Wu, Aparajeeta Majumder, Manthan N Patel, Sachchidanand Tiwari, Carolann L Espy, Fengyi Dong, Anit Shah, Vladimir V Shuvaev, Elizabeth D Hood, Liam S Chase, Drew Weissman, Jeremy B Katzen, David B Frank, Mariko L Bennett, Oscar A Marcos-Contreras, Jacob W Myerson, Vladimir R Muzykantov, Sahily Reyes-Esteves, Jacob S Brenner: Targeting DNA-LNPs to Endothelial Cells Improves Expression Magnitude, Duration, and Specificity. bioRxiv Page: 2025.07.09.663747, October 2025.

2c1 Emma R. Kotes, Sarah Woidill, Russell D'Aiello, Amina Khan, Jacob McCann, Mark Ramos, Francesco Gavazzi, Stephanie Keller, Keith Van Haren, Ali Fatemi, Florian Eichler, Joshua Bonkowsky, Jamie Fraser, Lisa Emrick, Omar Sherbini, Ashley Hackett, Jeilo Gauna, Dandre Amos, Jordan Goodman, Amena Smith Fine, Amanda Nagy, Seungil Lee, Nicole Page, Johanna Schmidt, Amy Pizzino, Kayla Muirhead, Mariko Bennett, Amy Waldman, Justine Shults, Laura Adang, Robert Grundmeier, Adeline Vanderver: Clinically Important Endpoints in Individuals With Leukodystrophy: A Multisite Study. Annals of the Child Neurology Society 3: 176-187, July 2025.

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