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

Pham V, Sertori F, Cassidy MM, Maguire JA, Waxman EA, French DL, King K, Zhao Z, Gelb MH, Wongkittichotee P, Hong X, Davidson BL, Ahrens-Nicklas RC: A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency. Mol Genet Metab 141(2), Feb 2024.

Amado DA, Robbins AB, Smith AR, Whiteman K, Chillon-Bosch G, Chen YH, Fuller JA, Izda A, Nelson S, Dichter AI, Monteys AM, Davidson BL: AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS. bioRxiv Page: 01.31.578314, Feb 2024.

Gall-Duncan T, Luo J, Jurkovic C-M, Fischer LA, Fujita K, Deshmukh AL, Harding RJ, Tran S, Mehkary M, Li V, Leib DE, Chen R, Tanaka H, Mason AG, Levesque D, Khan M, Razzaghi M, Prasolava T, Lanni S, Sato N, Caron M-C, Panigrahi GB, Wang P, Lau R, Castel AL, Masson J-Y, Tippet L, Turner C, Spies M, LaSpada AR, Campos EI, Curtis MA, Boisvert F-M, Faull RLM, Davidson BL, Nakamori M, Okazawa H, Wold MS, Pearson CE: Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability. Cell 186(22): 4898-4919, Oct 2023.

Jackson RJ, Keiser MS, Meltzer JC, Fykstra DP, Dierksmeier SE, Melloni A, Nakajima T, Tecedor L, Ranum PT, Carrell E, Chen YH, Holtman DM, Davidson BL, Hyman BT: APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease. bioRxiv Aug 2023 Notes: Preprint.

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, Izda A, Davidson BL: Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain. Mol Ther 31(3): 760-773, March 2023.

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.

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.

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Last updated: 06/12/2024
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