Beverly L. Davidson, Ph.D.

faculty photo
Professor of Pathology and Laboratory Medicine
Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia
Chief Scientific Strategy Officer (CSS), Children's Hospital of Philadelphia Research Institute
Department: Pathology and Laboratory Medicine
Graduate Group Affiliations

Contact information
The Children's Hospital of Philadelphia
3501 Civic Center Boulevard, 5060 CTRB
Philadelphia, PA 19104
Office: 267-426-0929
Fax: 215-590-3660
Education:
B.S. (Biology Major/Chemistry Minor; High Distinction)
Nebraska Wesleyan University, 1981.
Ph.D. (Biological Chemistry)
University of Michigan, 1987.
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Description of Research Expertise

Keywords:
Neurodegenerative Disease
RNA biology
Gene therapy
Animal models
Human treatment

Our lab is focused on genetic diseases that affect the brain. We study the mechanisms by which mutant gene products contribute to disease, and why certain brain regions are more susceptible. The team employs advanced molecular methods, sequencing and imaging modalities in animal models. The lab is also engaged in the development of next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments.

Selected Publications

Devinsky O, Coller J, Ahrens-Nicklas R, Liu XS, Ahituv N, Davidson BL, Bishop KM, Weiss Y, Mingorance A: Gene therapies for neurogenetic disorders. Trends Mol Med 17: S1471-4919(25)00015-2, Feb 2025 Notes: DOI: 10.1016/j.molmed.2025.01.015.

Nguyen L, Ajredini R, Shu G, Romano LEL, Tomas RF, Bell LR, Ranum PT, Zu T, Banez Coronel M, Kelley CP, Redding-Ochoa J, Nizamis E, Milloni A, Connors TR, Gaona A, Thangaraju K, Pletnikova O, Clark HB, Davidson BL, Yachnis AT, Golde TE, Lou XY, Wang ET, Renton AE, Goate A, Valdmanis PN, Prokop S, Troncoso JC, Hyman BT, Ranum LPW: CASP8 intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer's disease risk. Proc Natl Acad Sci USA 122(7): e2416885122, Feb 2025.

O'Driscoll E, Arora S, Lang JF, Davidson BL, Shalem O: CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production. Mol Ther Methods Clin Dev 33(1): 101408, Jan 2025 Notes: doi: 10.1016/j.omtm.2025.101408.

Carrell ST, Carrell EM, Giovenco RC, Davidson BL: Co-opting MBNL-dependent alternative splicing cassette exons to control gene therapy in myotonic dystrophy. bioRxiv Jan 2025 Notes: https://doi.org/10.1101/2025.01.23.634565.

O'Driscoll EE, Arora S, Lang JF, Davidson BL, Shalem O: CRISPR screen for rAAV production implicates genes associated with infection. bioRxiv Page: 09.17.613356, Sept 2024 Notes: doi: 10.1101/2024.09.17.613356.

Fagan KJ, Chillon G, Carrell EM, Waxman EA, Davidson BL: Cas9 editing of ATXN1 in a spinocerebellar ataxis type 1 mice and human iPSC-derived neurons. Mol Ther Nucleic Acids 35(4): 102317, Aug 2024.

Felix AJ, Wilson T, Randell R, Marotta N, Uchida K, Boland MJ, Davidson BL, Prosser BL: Generation of humanized mouse models to support therapeutic development for SYNGAP1 and STXBP1 disorders. bioRxiv Page: 08.22.609238, Aug 2024 Notes: doi: 10.1101/2024.08.22.609238.

Liu X, Jean-Gilles R, Baginski J, Cai C, Yan R, Zhang L, Lance K, van der Loo JCM, Davidson BL: Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization. Mol Ther Methods Clin Dev 32(3): 101298, July 2024.

Tecedor L, Chen Y, Leib DE, Ranum PT, Simpson BP, Keiser MG, McFadden M, Mackiewicz A, Lewandowski B, Lysenko E, Cha J-H, Davidson BL: AAV-EP+ in Preclinical Studies for Late Infantile Neuronal Ceroid Lipofuscinosis. Presented at the 27th Annual Meeting of the American Society for Gene and Cell Therapy, Baltimore, MD May 2024.

Romano LEL, Preston C, Ortiz T, Ajeridini R, Golliher H, Carrell E, Keiser M, Ames A, Davidson BL, Ranum LPW: AAV-Based anti-RAN Antibody Therapy for C9orf72 ALSFTD. Presented at the 27th Annual Meeting of the American Society for Gene and Cell Therapy, Baltimore, MD May 2024.

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Last updated: 02/26/2025
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