Beverly L. Davidson, PhD

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

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

• Samelson-Jones BJ, Martos-Rus, C, Yrigollen CM, Tecedor L, Small JC, Matesanz SE, Watson CT, Vanden Huevel AR, Carrig S, Yum SW, Brandsema JF, Lin K, Wittlieb-Weber C, Viaene AN, Russo PP, Partington S, Huynh K, Seneviratne T, Juarez Rojas S, Doshi BS, Chieh P, Flanigan KM, Frair E, Nicolau S, Lawlor MW, Crudele JM, Davidson BL, George JA : Cardiotoxic effects and microdystrophin expression after gene therapy for DMD N Engl J Med 395(6) : 615-617, Aug 2026
• Felix AJ, Brown B, Marotta N, Gessner MJ, Houserova M, Huerta-Ocampa I, Wilson T, Randell R, Dawicki-McKenna J, Reinhardt D, Uchida K, McSalle I, McKee J, Helbig I, Boland MJ, Davidson BL, Prosser BL : Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder Mol Psychiatry Jul 2026 Notes: doi: 10.1038/s41380-026-03698. Online ahead of print.
• Ahrens-Nicklas RC, Kotch C, Roche AM, Everett JK, Reddy S, Santi M, Madsen PJ, Martos-Rus C, Yrigollen CM, Small JC, McCague S, Davidson BL, Samelson-Jones BJ, Surrey LF, Li M, Ficicioglu C, Wang R, Bushman FD, George LA : Neuroepithelial tumor with AAV integration after ICM vector delivery N Eng J Med 394(21) : 2126-2133, June 2026
• Cooney A, Chen YH, Lewandowski BC, Lamer S, Boysen G, Kulhankova K, Vu A, Newase P, Sinn P, Davidson B, McCray PB : Optimized AAV capsids robustly transduce airway epithelial cells. bioRxiv May 2026 Notes: doi: https://doi.org/10.64898/2026.05.10.723853
• Feierman ER, Whittaker MN, Quigley A, Brooks DL, McVeigh P, Nan AX, Hsu A, Said H, Soliman OY, Giovenco R, Davidson BL, Alameh MG, Liu DR, Wang X, Musunuru K, Ahrens-Nicklas RC : Implications of the FDA’s new plausible mechanism framework for the development of a personalized in vivo prime editing platform Am J Hum Genet 113(5) : 891-899, May 2026
• Robbins AB, Ranum PT, Huerto-Ocampo I, Kuckyr M, Davidson BL : Temporal single-cell atlas of full-length Huntington’s disease mouse model defines stage-specific signatures of corticostriatal dysfunction. Mol Neurodegener 28(1) : 41, May 2026
• Carrell ST, Carrell EM, Giovenco R, Davidson BL : Co-Opting MBNL-Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy Ann Neurol 99(1) : 211-222, Jan 2026 Notes: doi: 10.1002/ana.78024. Epub ahead of print.
• Simpson BP, Ranum PT, Leib DE, Tecedor L, Giovenco RC, Huerta-Ocampo I, Hudley AR, Smith N, Fluta CM, Cali CP, Benoit J, Soper JC, Connelly J, Yohrling GJ, Ghoroghchian PP, Cha JH, Davidson BL : Computational modeling and preclinical validation support targeting somatic instability for Huntington’s disease treatment bioRxiv Jan 2026 Notes: doi: https://doi.org/10.64898/2026.01.06.697909
• Robbins AJ, Ranum PT, Huerta-Ocampo I, Kuckyr M, Davidson BL : Temporal single-cell atlas of full-length Huntington’s disease mouse model defines stage-specific signatures of corticostriatal dysfunction bioRxiv Jan 2026 Notes: doi: https://doi.org/10.64898/2026.01.07.698249
• Chamberlain JS, Davidson BL, George LA, Bryne BJ, Barrett D : The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve Mol Ther 33(10) : 4694-4695, Oct 2025