ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026
Microglia replacement by peripheral delivery of CSF1R inhibitor-resistant hematopoietic cells.
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Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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4citing papers in PubMed
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4 citing papers in PubMed.
- Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
- Microglia and neuroinflammation: An in-depth analysis from functional diversity to disease mechanisms.Clinical and translational medicine · 2026Review
- CSF1R-related leukoencephalopathy: experimental models and potential for treatment.Disease models & mechanisms · 2026Review
- The senescent niche hypothesis: microglial dysfunction and replacement strategies in drug-resistant epilepsy.Frontiers in immunology · 2026Review
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Authors and funding
30 authors.
Sonia I LombrosoDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania, Philadelphia, PA 19104, USA.
Emily I ChurchDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Ryan N AndersonDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Systems Pharmacology and Translational Therapeutics, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Carleigh A O'BrienDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Yixuan ZhuDepartment of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Asif A DarDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Freddy S PurnellEpigenetics Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Biology, School of Arts and Sciences, University of Pennsylvania, Philadelphia, PA 19104, USA.
Eli M LevittDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Brian TemsamritDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Fazeela YaqoobDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
William H AisenbergDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mara DavisCenter for Cellular Immunotherapies, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA.
Bilal ElfayoumiDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Sai ChaluvadiDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Neuroscience, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Kelsey M NemecDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Neuroscience, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Jessie AxsomDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Daniel A ShapiroDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Chet Huan OonDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Center for Cellular Immunotherapies, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA.
Jean Paul ChadarevianDepartment of Neurobiology and Behavior, University of California, Irvine, Irvine, CA 92697, USA; Institute for Memory Impairments and Neurological Disorders, University of California, Irvine, Irvine, CA 92697, USA; Sue and Bill Gross Stem Cell Research Center, University of California, Irvine, Irvine, CA 92697, USA.
Charles-Antoine AssenmacherComparative Pathology Core, Department of Pathobiology, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
Stefano De ArcangeliDepartment of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Esha BanerjeeComparative Pathology Core, Department of Pathobiology, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
Sangeeta ShuklaDepartment of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Hayk DavtyanInstitute for Memory Impairments and Neurological Disorders, University of California, Irvine, Irvine, CA 92697, USA; Sue and Bill Gross Stem Cell Research Center, University of California, Irvine, Irvine, CA 92697, USA.
Will BailisDepartment of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Christopher A HunterDepartment of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mathew Blurton-JonesDepartment of Neurobiology and Behavior, University of California, Irvine, Irvine, CA 92697, USA; Institute for Memory Impairments and Neurological Disorders, University of California, Irvine, Irvine, CA 92697, USA; Sue and Bill Gross Stem Cell Research Center, University of California, Irvine, Irvine, CA 92697, USA.
Mariko L BennettDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Michael S HaneyDepartment of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
F Chris BennettDepartment of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: frederick.bennett@pennmedicine.upenn.edu.
Funding
UNIVERSITY OF PENNSYLVANIA CAN CTR SUPPORT GRANTP30CA016520 · NCI · UNIVERSITY OF PENNSYLVANIA · PI Robert H. Vonderheide · 1985 to 2026
$222.3MMEDICAL SCIENTIST TRAINING PROGRAMT32GM007170 · NIGMS · UNIVERSITY OF PENNSYLVANIA · PI BRASS, LAWRENCE F · 1985 to 2022
$54.0MIMMUNOBIOLOGY OF NORMAL AND NEOPLASTIC LYMPHOCYTEST32CA009140 · NCI · UNIVERSITY OF PENNSYLVANIA · PI Malay Haldar, WARREN S PEAR · 1985 to 2026
$16.1MTraining in Alzheimer’s and Age-Related Neurodegenerative DiseasesT32AG000255 · NIA · UNIVERSITY OF PENNSYLVANIA · PI ALICE S CHEN-PLOTKIN, VIRGINIA M LEE · 1997 to 2026
$12.5MPREDOCTORAL TRAINING PROGRAM IN PHARMACOLOGYT32GM008076 · NIGMS · UNIVERSITY OF PENNSYLVANIA · PI BLENDY, JULIE A · 1985 to 2023
$10.8MTraining in the Neurobiology of Aging and Alzheimers DiseaseT32AG000096 · NIA · UNIVERSITY OF CALIFORNIA-IRVINE · PI VIVEK SWARUP, Craig E Stark · 1985 to 2026
$9.6MTraining Program in Neurodevelopmental DisabilitiesT32NS007413 · NINDS · CHILDREN'S HOSP OF PHILADELPHIA · PI AMELIA J EISCH, ERIC D MARSH · 1998 to 2026
$8.9MSCHIZOPHRENIA: A NEUROPSYCHIATRIC PERSPECTIVET32MH019112 · NIMH · UNIVERSITY OF PENNSYLVANIA · PI GUR, RAQUEL E · 1990 to 2024
$6.2MNeuronal Latency and ToxoplasmaR01AI157247 · NIAID · UNIVERSITY OF PENNSYLVANIA · PI CHRISTOPHER A HUNTER, Anita Koshy · 2020 to 2026
$4.7MStress-modified growth signaling in dynamic nutrient environmentsR35GM138085 · NIGMS · CHILDREN'S HOSP OF PHILADELPHIA · PI Will H. Bailis · 2020 to 2026
$2.7MRole of Brain Macrophages in the Pathogenesis and Treatment of Globoid Cell LeukodystrophyR01NS120960 · NINDS · UNIVERSITY OF PENNSYLVANIA · PI BENNETT, FREDERICK · 2021 to 2025
$2.3MUnlocking microglia targeting for neurotherapeuticsDP5OD036159 · OD · CHILDREN'S HOSP OF PHILADELPHIA · PI Mariko L. Bennett · 2023 to 2026
$2.2MNCI NIH HHS P30 CA016520NCI NIH HHS T32 CA009140NIAID NIH HHS R01 AI157247NIA NIH HHS T32 AG000096NIA NIH HHS T32 AG000255NIA NIH HHS T32 AG073088NIGMS NIH HHS R35 GM138085NIGMS NIH HHS T32 GM007170NIGMS NIH HHS T32 GM008076NIH HHS DP5 OD036159NIMH NIH HHS T32 MH019112NINDS NIH HHS R01 NS120960NINDS NIH HHS R01 NS138537NINDS NIH HHS T32 NS007413
Abstract
Microglia replacement holds great promise for the study and treatment of neurological diseases. We previously achieved high-efficiency replacement by engineering an inhibitor-resistant human CSF1R variant, G795A. Here and in Chadarevian et al, we introduce a transgenic mouse line carrying the homologous murine inhibitor resistance mutation, G793A. G793A confers resistance to CSF1R inhibitors (CSF1Ris) without impacting cell or organismal function, allowing brain-wide microglia replacement after intravascular delivery of myeloid cells or progenitors. G793A macrophages have normal CSF1R signaling and effector responses in cell-based assays. CSF1Ri treatment paired with peripheral vascular delivery of G793A myeloid cells, including ex vivo expanded hematopoietic stem cells, leads to microglia replacement in neonates and adults, with low peripheral chimerism. With vascular adoptive transfer of GFP
Indexed as
Hematopoietic Stem CellsHematopoietic Stem Cell TransplantationMicrogliaReceptors, Granulocyte-Macrophage Colony-Stimulating FactorAnimalsHumansMiceMice, TransgenicCsf1r protein, mouseReceptors, Granulocyte-Macrophage Colony-Stimulating FactorCSF1Rmicrogliamicroglia replacementmyeloid cell therapiesneuroimmunologystem cell transplantation
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.