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

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 2018 (in press).

Keiser MK, Chen YH, Davidson BL: Techniques for intracranial stereotoxic injections of adeno-associated viral vectors in adult mice. Curr Protoc Mouse Biol Page: e58, Nov 2018 Notes: doi: 10.1002/cpmo.58.

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.

Chen YH, Keiser MS, Davidson BL: Adeno-associated virus production, purification and titering. Curr Protoc Mouse Biol Page: e:56, Nov 2018 Notes: doi: 10.1002/cpmo.56.

Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tift CJ: Lysosomal storage diseases. Nat Rev Dis Primers 4(1): 27, Oct 2018 Notes: doi: 10.1038/s41572-018-0025-4.

Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ: Lysosomal storage diseases (author correction). Nat Rev Dis Primers 4(1): 36, Oct 2018 Notes: Erratum in: Author Correction: Lysosomal storage diseases. [Nat Rev Dis Primers. 2018]

Cheng C, Spengler RM, Keiser MS, Monteys AM, Riedeers JM, Ramachandran S, Davidson BL: The long noncoding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease-gene dependent toxicities. Hum Mol Genet Sept 2018 Notes: doi:10.1093/hmg/ddy331.

Sun JH, Zhou L, Emerson DJ, Phyo SA, Titus KR, Gong W, Gilgenast TG, Beagan JA, Davidson BL: Disease-associated short tandem repeats co-localize with chromatin domain boundaries. Cell 175(1): 224-238, August 2018.

Chen YH, Zheng S, Tecedor L, Davidson BL: Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy. Mol Ther 26(4): 1118-1126, April 2018 Notes: pii: S1525-0016(18)30017-0; (Epub ahead of print].

Schultz ML, Tecedor L, Lysenko E, Ramachandran S, Stein CS, Davidson BL: Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivo. Neurobiol Dis 115: 182-193, April 2018 Notes: doi: 10.1016/j.nbd.2018.04.010. [Epub ahead of print]

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Last updated: 12/19/2018
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