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Beverly L. Davidson, Ph.D.
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Professor of Pathology and Laboratory Medicine
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Department: Pathology and Laboratory Medicine
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The Children's Hospital of Philadelphia
49 3501 Civic Center Boulevard, 5060 CTRB
Philadelphia, PA 19104
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49 3501 Civic Center Boulevard, 5060 CTRB
Philadelphia, PA 19104
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Office: 267-426-0929
3e Lab: 267-425-2162
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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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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.
12d 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 Aug 2025 Notes: doi: 10.1002/ana.78024. Epub ahead of print.
169 Amado DA, Robbins AB, Whiteman KR, Smith AR, Chillon G, Chen Y, Fuller JA, Patty NA, Izda A, Cheng C, Nelson S, Dichter AI, Mazzone EO, Montey AM, Davidson BL: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice. Nat Commun 16(1): 5334, June 2025.
c7 Giovenco R, Carrell E, Nelson S, Tecedor L, Lysenko E, Keiser M, Davidson B: Delivery of miRNA by Self-Complimentary AAV Provides ATXN1 Knockdown in c8 SCA1 Mice and Supports Therapeutic Translation Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #495), May 2025.
166 Cooney A, Chen Y, Lewandowski B, Lamar S, Newase P, Kulhankova K, Davidson B, McCray P: Peptide insertions enhance AAV capsid tropism for airway epithelia. Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #1523), May 2025.
d2 Leib D, Villa K, Tecedor L, McFadden M, Mackiewicz A, Yohrling G, Davidson B, Cha J-H: A Non-GLP Dose Range Finding Study in Cynomolgus Macaques Evaluating the 56 Biodistribution, Expression, and Safety of LTS-101, a Novel Preclinical Gene Therapy cc Candidate for the Treatment of CLN2 Batten Disease Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #521), May 2025.
b9 Lingren J, Chen Y, Lewandowski B, Davidson B, Sabatino D: Identification of bioengineered AAV capsids with enhanced targeting of liver be sinusoidal endothelial cells (LSECs) Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #511), May 2025.
94 Kuckyr M, Amado D, Davidson B: Base Editing Premature Stop Codons Within the ATXN2 Repeat Reduces ab Pathogenic Polyglutamine Proteins. Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy(Abstract #1103), May 2025.
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Selected Publications
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.
12d 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 Aug 2025 Notes: doi: 10.1002/ana.78024. Epub ahead of print.
169 Amado DA, Robbins AB, Whiteman KR, Smith AR, Chillon G, Chen Y, Fuller JA, Patty NA, Izda A, Cheng C, Nelson S, Dichter AI, Mazzone EO, Montey AM, Davidson BL: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice. Nat Commun 16(1): 5334, June 2025.
c7 Giovenco R, Carrell E, Nelson S, Tecedor L, Lysenko E, Keiser M, Davidson B: Delivery of miRNA by Self-Complimentary AAV Provides ATXN1 Knockdown in c8 SCA1 Mice and Supports Therapeutic Translation Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #495), May 2025.
166 Cooney A, Chen Y, Lewandowski B, Lamar S, Newase P, Kulhankova K, Davidson B, McCray P: Peptide insertions enhance AAV capsid tropism for airway epithelia. Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #1523), May 2025.
d2 Leib D, Villa K, Tecedor L, McFadden M, Mackiewicz A, Yohrling G, Davidson B, Cha J-H: A Non-GLP Dose Range Finding Study in Cynomolgus Macaques Evaluating the 56 Biodistribution, Expression, and Safety of LTS-101, a Novel Preclinical Gene Therapy cc Candidate for the Treatment of CLN2 Batten Disease Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #521), May 2025.
b9 Lingren J, Chen Y, Lewandowski B, Davidson B, Sabatino D: Identification of bioengineered AAV capsids with enhanced targeting of liver be sinusoidal endothelial cells (LSECs) Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, New Orleans, LA(Abstract #511), May 2025.
94 Kuckyr M, Amado D, Davidson B: Base Editing Premature Stop Codons Within the ATXN2 Repeat Reduces ab Pathogenic Polyglutamine Proteins. Presented at the 28th Annual Meeting of the American Society of Gene and Cell Therapy(Abstract #1103), May 2025.
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