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
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

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

Carrell EM, Chen YH, Ranum PT, Coffin SL, Singh LN, Tecedor L, Keiser MG, Hudry E, Hyman BT, Davidson BL: VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF. Mol Ther Nucleic Acids 33: 296-304, July 2023.

Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Maguire JA, Gagne AL, Heller EA, French DL, Davidson, BL, Prosser, BL: Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching. Nat Commun 14(1): 2628, May 2023.

Simpson, BP, Yrigollen CM, Izea A, Davidson BL: Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain. Mol Ther 31(3): 760-773, March 2023 Notes: DOI: 10.1016/j.ymthe.2023.01.004.

Ranum PT, Tecedor L, Keiser MS, Chen YH, Leib DE, Liu X, Davidson, BL: Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates. Mol Ther 31(3): 609-612, March 2023 Notes: doi: 10.1016/j.ymthe.2022.12.018.

Fang L, Monteys AM, Durr A, Keiser M, Cheng C, Harapanahalli A, Gonzalez-Alegre P, Davidson BL, Wang K: Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing. HGG Adv 4(1): 100146, Sept 2022 Notes: Erratum: HGG Adv 2023 Jun 20;4(3)100212.

St-Cyr S, Child DD, Giaime E, Smith AR, Pascua CJ, Hahm S, Saiah E, Davidson BL: Huntington's disease phenotypes are improved via mTORC1 modulation by small molecule therapy. PLoS One 17(8): e0273710, August 2022 Notes: doi: 10.1371/journal.pone.0273710.

Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J (American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration): Evaluating the state of the science for adeno-associated virus integration: An integrated perspective. Mol Ther 30(8): 2646-2663, Aug 2022.

Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Heller EA, French DL, Davidson BL, Prosser BL: Mapping PTBP splicing in human brain identifies targets for therapeutic splice switching SNYGAP1. bioRxiv July 2022 Notes: doi:

Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bonnemann C, Buxbaum JD, Corcoran G, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CF, Sahin M, (Gene Therapy Workshop Faculty): Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther 30(7): 2416-2428, July 2022.

Yrigollen CM, Simpson B, Lim E, Chen YH, Davidson B: The molecular and behavioral phenotypes altered in CGG knock-in mice edited using CRISPR. Presented at the 25th annual meeting of the American Society for Gene and Cell Therapy, Washington, DC Page: Poster M-160, May 2022.

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Last updated: 08/24/2023
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