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

Ahrens-Nicklas RC, Tecedor L, Hall A, Lysenko E, Cohen AS, Davidson BL, Marsh ED: Neuronal network dysfunction precedes storage and neurodegeneration in a lysosomal storage disorder. JCI Insight Page: pii: 131961, Oct 2019 Notes: https://doi.org/10.1172/jci.insight.131961.

Davidson BL, Arruda VR: Gene therapy matures to medicines. Hum Mol Genet 28(R1): R1-R2, Oct 2019 Notes: doi:10.1093/hmg/ddz182.

Davidson BL: Doubling down on siRNAs in the brain. Nat Biotechnol 37(8): 865-866, Aug 2019.

Lang JF, Toulmin SA, Brida KL, Eisenlohr LC, Davidson BL: Standard screening methods underreport AAV-mediated transduction and gene editing. Nat Commun 10(1): 3415, July 2019.

Simpson BP, Davidson BL: CRISPR-Cas gene editing for neurological disease. Nervous System Drug Delivery. Lonser R, Sarntinoranont M, Bankiewicz K (eds.). Academic Press, Elsevier,(Chapter 18), 365-376, June 2019 Notes: 1st Edition.

Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ: Publisher Correction: Lysosomal storage diseases. Nat Rev Dis Primers 5(1): 34, May 2019.

Amado DA, Rieders JM, Diatta F, Hernandez-Con P, Singer A, Zhang J, Lancaster E, Davidson BL, Chen-Plotkin AS: AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated hippocampal degeneration. Mol Ther 27(2), Feb 2019.

Maguire JA, Gagne AL, Gonzalez-Alegre P, Davidson BL, Shakkottai V, Gadue P, French DL.: Generation of a Spinocerebellar Ataxia Type 2 induced pluripotent stem cells. CHOPi002-A and CHOPi003-A. from patients with abnormal CAG repeats in the coding region of the ATXN2 gene. Stem Cell Res 34: 101361, Jan 2019.

Yrigollen CM, Davidson BL: CRISPPR to the rescue: advances in gene editing for the FMR1 gene. Brain Sci 9(1): pii:E17, Jan 2019.

Chen YH, Keiser MS, Davidson BL: Viral vectors for gene transfer. Curr Protoc Mouse Biol Page: E58, Nov 2018 Notes: doi: 10.1002/cpmo.58.

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Last updated: 10/25/2019
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