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Beverly L. Davidson, Ph.D.
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Professor of Pathology and Laboratory Medicine
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Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia
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Chief Scientific Strategy Officer (CSS), Children's Hospital of Philadelphia Research Institute
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Department: Pathology and Laboratory Medicine
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Contact information
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The Children's Hospital of Philadelphia
4a 3501 Civic Center Boulevard, 11056 CTRB
Philadelphia, PA 19104
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4a 3501 Civic Center Boulevard, 11056 CTRB
Philadelphia, PA 19104
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Office: 267-426-0929
3e Lab: 267-425-2162
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Email:
davidsonbl@chop.edu
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davidsonbl@chop.edu
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Education:
21 9 B.S. 3c (Biology Major/Chemistry Minor; High Distinction) c
35 Nebraska Wesleyan University, 1981.
21 a Ph.D. 21 (Biological Chemistry) c
2f University of Michigan, 1987.
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Permanent link21 9 B.S. 3c (Biology Major/Chemistry Minor; High Distinction) c
35 Nebraska Wesleyan University, 1981.
21 a Ph.D. 21 (Biological Chemistry) c
2f University of Michigan, 1987.
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23 Neurodegenerative Disease
15 RNA biology
16 Gene therapy
18 Animal models
19 Human treatment
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209 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.
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Description of Research Expertise
1a Keywords:23 Neurodegenerative Disease
15 RNA biology
16 Gene therapy
18 Animal models
19 Human treatment
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209 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.
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1be Prosser B, Felix A, Brown B, Marotta N, Gessner M, Houserova M, Huerta-Ocampa I, Wilson T, Randell R, Dawicki-McKenna J, Reinhardt D, Uchida K, McSalle I, McKee J, Helbig I, Davidson BL: Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder. Mol Psychiatry May 2026 Notes: doi: 10.1056/NEJMoa2601608. Online ahead of print.
184 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. New Eng J Med May 2026.
146 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.
1d1 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.
15e 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.
13d 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.
187 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.
1ec Sichlinger L, Reilly MB, Arora S, Zhang S, Marotta N, Rodriguez-Acevedo KL, Hooks M, Czarnecki KS, Winter JJ, Waxman EA, Dungan LV, Hong I, Araki Y, Johnson R, Huganir RL, Pavani G, French DL, Davidson BL, Prosser BL, Heller EA: CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability. bioRxiv Oct 2025 Notes: doi: 10.1101/2025.10.28.685100.
17c Amado DA, Robbins AB, Whiteman KR, Smith AR, Chillon G, Chen Y, Fuller JA, Patty NA, Izda A, Cheng C, Nelson S, Dichter AI, Mazzoni EO, Monteys AM, Davidson BL: Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice. Nat Commun 16(1): 9356, Oct 2025.
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Selected Publications
1a9 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 2026.1be Prosser B, Felix A, Brown B, Marotta N, Gessner M, Houserova M, Huerta-Ocampa I, Wilson T, Randell R, Dawicki-McKenna J, Reinhardt D, Uchida K, McSalle I, McKee J, Helbig I, Davidson BL: Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder. Mol Psychiatry May 2026 Notes: doi: 10.1056/NEJMoa2601608. Online ahead of print.
184 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. New Eng J Med May 2026.
146 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.
1d1 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.
15e 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.
13d 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.
187 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.
1ec Sichlinger L, Reilly MB, Arora S, Zhang S, Marotta N, Rodriguez-Acevedo KL, Hooks M, Czarnecki KS, Winter JJ, Waxman EA, Dungan LV, Hong I, Araki Y, Johnson R, Huganir RL, Pavani G, French DL, Davidson BL, Prosser BL, Heller EA: CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability. bioRxiv Oct 2025 Notes: doi: 10.1101/2025.10.28.685100.
17c Amado DA, Robbins AB, Whiteman KR, Smith AR, Chillon G, Chen Y, Fuller JA, Patty NA, Izda A, Cheng C, Nelson S, Dichter AI, Mazzoni EO, Monteys AM, Davidson BL: Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice. Nat Commun 16(1): 9356, Oct 2025.
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