Beverly L. Davidson, Ph.D.

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Professor of Pathology and Laboratory Medicine
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

Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction, with a focus on:

i) recessive, childhood onset neurodegenerative disease, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease;
ii) dominant genetic diseases, specifically the CAG repeat disorders, Huntington’s disease and spinal cerebellar ataxia;
iii) understanding how changes in the transcriptome impact neural development and neurodegenerative disease processes.

Our research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule or gene therapy based strategies for therapy. In recent work, we demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. We continue to develop novel vector systems to improve therapeutic outcomes.

Therapies for dominant disorders are an exciting challenge and require that the dominant disease allele be silenced. To approach this, we developed reagents for expressing inhibitory RNAs or editing machinery (e.g., CrispR/Cas9 approaches) in vivo to improve disease phenotypes in relevant animal models.

Finally, we investigate how the transcriptome is altered in neurological diseases. Evaluation of splicing changes has led us to discover novel players in disease pathogenesis that include noncoding RNAs and RNA binding proteins. This work is revealing new pathways of pathogenesis and novel targets for therapy.

Selected Publications

Keiser MS, Ranum PT, Yrigollen CM, Carrell EM, Smith GR, Muehlmatt AL, Chen YH, Stein, JM, Wolf RL, Radaelli E, Lucas TJ2nd, Gonzalez-Alegre P, Davidson BL: Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain. Nat Med Oct 2021 (in press) Notes: DOI: 10.1038/s41591-021-01522-3.

Morris JA, Boshoff CH, Schor NF, Wong LM, Gao G, Davidson BL: Next Generation Strategies for Gene-Targeted Therapies of Central Nervous System Disorders: A Workshop Summary. Mol Ther Page: S1525-0016(21)00195-7, Sept 2021 (in press) Notes: doi: 10.1016/j.ymthe.2021.09.010.

Akimov SS, Jiang M, Kedaigle AJ, Arbez N, Marque LO, Eddings CR, Ranum PT, Whelan E, Tang A, Wang R, DeVine LR, Talbot CC, Cole RN, Ratovitski T, Davidson BL, Fraenkel E, Ross CA: Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics. Hum Mol Genet July 2021 (in press) Notes: doi: 10.1093/hmg/ddab200

Prezelski K, Keiser M, Stein J, Lucas TH, Davidson B, Gonzalez P, Vitale F: Design and validation of a multi-point injection technology for MR-guided convection enhanced delivery in the brain. Front Med Technol 2021 (in press).

Amado DA, Davidson BL: Gene therapy for ALS: A review. Mol Ther Page: S1525-0016(21)00195-7, April 2021 Notes: doi: 10.1016/j.ymthe.2021.04.008.

Monteys AM, Hundley AA, Ranum PT, Tecedor L, Muehlmatt A, Lim E, Lukashev, Sivasankaran R, Davidson BL : Regulated control of gene therapies by drug-induced splicing. Nature 596(7871), August 2021 Notes: DOI: 10.1038/s41586-021-03770-2 Epub 2021 July 28.

Byrne BJ, McCall AL, Davidson BL, Samulski RJ: Mavis Agbandje-McKenna's Lifelong Commitment to Teaching and Research. Hum Gene Ther 32(7-8): 319-320, April 2021 Notes: doi: 10.1089/hum.2021.29157.alm.

Pluciennik A, Liu Y, Molotsky E, Marsh GB, Ranxhi B, Arnold FJ, St-Cyr S, Davidson BL, Pourshafie N, Lieberman AP, Gu W, Todi SV, Merry DE: Deubiquitinase USP7 contributes to the pathogenicity of spinal and bulbar muscular atrophy. J Clin Invest 131(1): :e134565, Jan 2021 Notes: https://doi.org/10.1172/JCI134565.

Morozko EL, Smith-Geater C, Monteys AM, Pradhan S, Lim RG, Langfelder P, Kachemov M, Hill A, Stocksdale JT, Cullis PR, Wu J, Ochaba J, Miramontes R, Chakraborty A, Hazra TK, Lau A, St-Cyr S, Orellana I, Kopan L, Wang KQ, Yeung S, Leavitt BR, Reidling JC, Yang XW, Steffan JS, Davidson BL, Sarkar PS, Thompson LM.: PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington's disease. Proc Natl Acad Sci USA 118(4), Jan 2021 Notes: doi: 10.1073/pnas.2021836118.

Victor MB, Richner M, Olsen HE, Lee SW, Monteys AM, Ma C, Huh CJ, Zhang B, Davidson BL, Yang XW, Yoo AS: Author Correction: Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes. Nat Neurosci 23(10): 1307, Oct 2020 Notes: 10.1038/s41593-020-00714-3.

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Last updated: 10/25/2021
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