Beverly L. Davidson, PhD

Professor of Pathology and Laboratory Medicine
Professor of Genetics
Perelman School of Medicine at the University of Pennsylvania 
Chief Scientific Strategy Officer at Children’s Hospital of Philadelphia
Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics
Arthur V. Meigs Chair in Pediatrics at CHOP

Contact InformationChildren's Hospital of Philadelphia
3501 Civic Center Boulevard, 5060 CTRB
Philadelphia, PA 19104

Office: 267-426-0929
Fax: 215-590-3660


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.


B.S. (Biology Major/Chemistry Minor; High Distinction), Nebraska Wesleyan University, 1981
Ph.D. (Biological Chemistry), University of Michigan, 1987

Specialty Certification

Postgraduate Training

Research Assistant, University of Nebraska, Nebraska, 1982-1983
Postdoctoral Fellow, University of Michigan, Michigan, 1988-1988
Research Investigator, University of Michigan, Michigan, 1990-1992
Assistant Research Scientist, University of Michigan, Michigan, 1992-1994

Awards and Honors

President Scholarship Award, 1977-1981
Beta Beta Beta Biology Honorary Society, 1979
Phi Kappa Phi Honor Society, 1980
VI International Symposium on Human Purine and Pyrimidine Metabolism, 1988
Fellow, American Association for the Advancement of Science, 2006
Regents Faculty Excellence Award, 2007
Carver Research Program of Excellence, University of Iowa, 2007-2014
Mathilde Solowey Award, National Institutes of Health, 2008
Eureka Award, National Institutes of Health, 2008
SJ DeArmond Lecture, American Association of Neuropathologists, 2011
Distinguished Graduate Lecture, University of Michigan Dept of
Biological Chemistry, Ann Arbor, MI, 2011
Innovator Award, University of Iowa, 2012
Inventor Award, University of Iowa, 2013
Arthur V. Meigs Chair in Pediatrics, The Children's Hospital of Philadelphia, 2014-Present
Recipient, The Leslie Gehry Prize for Innovation in Science, Hereditary Disease Foundation, 2015
Member, American Academy of Arts and Sciences, 2017
Member, International Selection Committee, 2018 Bower Award for Achievement in Science, The Franklin Institute, 2017-2018
FE Bennett Memorial Lectureship Award, American Neurological Association, 2018
Member, National Academy of Medicine, 2019

Memberships and Professional Organizations

Mental Retardation Research Committee, NICHD, 1995 - 1999
NIH Study Sections, 1995 - Present
American Association for the Advancement of Science (AAAS), 1995 - Present
National Gene Vector Laboratory Scientific Review Board, 1996 - 2007
American Society for Gene and Cell Therapy, 1997 - Present
Batten Disease Support and Research Association, Scientific Advisory Board, 2000 - Present
Hunters Hope, Scientific Advisory Board, 2000 - 2002
Amytrophic Lateral Sclerosis Association, Study Section, 2000 - 2003
Scientific Advisory Board, Oxford Biomedica Inc, 2001 - 2006
TIGET Site Visit Team, Milan, Italy, 2002 - 2002
Sirna Therapeutics, 2004 - 2007
TIGET Site Visit Team, Milan, Italy, 2006 - 2006
Hereditary Disease Foundation, 2006 - 2009
Sirna Therapeutics, 2006 - 2008
Scientific Advisory Board, Scientific Committee, Comitato Telethon Fondazione, 2007 - 2010
Centre de Genomica Regulacio, Barcelona Spain, 2007 - Present
TIGET Site Visit Team, Milan, Italy, 2007 - 2007
Robert Packard Center for ALS Research at Johns Hopkins, 2007 - Present
Oregon National Primate Research Center, 2008 - Present
NCL Foundation, Hamburg, Germany, 2008 - Present
TIGET Site Visit Team, Milan, Italy, 2008 - 2008
Wellstone Muscular Dystrophy Center Scientific Advisory Committee,
Minneapolis, MN, 2008 - 2011
Electorate Nominating Committee, Medical Sciences, AAAS, 2009 - 2012
TIGEM Site Visit Team, Naples, Italy, 2009 - Present
MDRNA, 2009 - 2010
UMass, 2009 - Present
TIGET Site Visit Team, Milan, Italy, 2009 - 2009
Basal Ganglia Disorders Linnaeus Consortium, Lund, Sweden, 2009 - 2014
Marina Biotech, 2010 - 2012
Scientific Committee, Comitato Telethon Fondazione, 2010 - 2011
TAGS study section, NIH, 2011 - Present
BDSRA Scientific Advisory Board, 2011 - Present
TIGEM Site Visit Team, Naples, Italy, 2011 - 2011
Hereditary Disease Foundation, 2012 - 2015
nLIFE, 2012 - Present
Spark Therapeutics, 2013 - Present
NIH Common Fund, 2013 - Present
TIGET Site Visit Team, Milan, Italy, 2014 - 2014
Member, External Advisory Committee, Pathophysiology and Global Therapy for Krabbe Disease, Washington University School of Medicine, St. Louis, MO, 2014 - 2015
Member, National Advisory Neurological Disorders and Stroke Council, NIH, NINDS, 2014 - Present
Member, Blue Ribbon Panel for Review of Intramural Programs, NINDS, NIH, 2014 - Present
NIH Common Fund, 2014 - Present
Member, American Society of Human Genetics, Social Issues Committee, 2015 - Present
Member, Medical Research Advisory Bord (MRAB), National Ataxia Foundation, 2015 - Present
Member, Data Safety Monitoring Board, Intrathecal Administration of scAAV9/Jet-GAN for the Treatment of Giant Axonal Neuropathy (NIH Sponsored), 2015 - Present
Member, Scientific Advisory Board, Sarepta Therapeutics, Cambridge, A, 2015 - Present
Member, Scientific Advisory Board, Intellia Therapeutics, 2015 - Present
Member, Scientific Advisory Committee, Huntington Study Group, 2015 - 2018
Member, Advisory Board, Horizon 2020 Twinning 2015, European Commission, 2015 - Present
Member, Scientific Advisory Board, Friends of Telethon, Italia, 2016 - Present
Member, Working Group of Council on Diversity, NIH/NINDS, 2016 - Present
Member, Scientific Director Search Committee, NIH/NINDS, 2016 - Present
Member, Scientific Advisory Board, Anhui IsoTex (Yun Tai) Biotech, Inc., 2017 - Present
Member, Packard Center Board of Advisors, Johns Hopkins University, 2017 - Present
Member, International Selection Committee, 2018 Bower Award and Prize for Achievement in Science. Franklin Institute Awards., 2017 - 2018
Member, Scientific Advisory Board, STXBP1 Foundation, 2017 - Present
Member, The College of Physicians of Philadelphia, 2018 - Present
Member, Scientific Advisory Board, Homology Medicines, Inc., 2019 - Present
Member, Scientific Advisory Board, Prevail Therapeutics, 2019 - Present
Member, Scientific Advisory Board, Panorama Medicine, Inc., 2019 - Present

Web Links

Selected Publications

Neuronal network dysfunction precedes storage and neurodegeneration in a lysosomal storage disorder

Ahrens-Nicklas RC, Tecedor L, Hall A, Lysenko E, Cohen AS, Davidson BL, Marsh ED, JCI Insight, 2019, PMID:31573978

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Gene therapy matures to medicines

Davidson BL, Arruda VR, Hum Mol Genet 28(R1): R1-R2, 2019, PMID:31595292

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Doubling down on siRNAs in the brain

Davidson BL, Nat Biotechnol 37(8): 865-866, 2019, PMID:31375811

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Standard screening methods underreport AAV-mediated transduction and gene editing

Lang JF, Toulmin SA, Brida KL, Eisenlohr LC, Davidson BL, Nat Commun 10(1): 3415, 2019, PMID:31363095

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CRISPR-Cas gene editing for neurological disease.

Simpson BP, Davidson BL, Nervous System Drug Delivery, 2019

Publisher Correction: Lysosomal storage diseases

Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ, Nat Rev Dis Primers 5(1): 34, 2019, PMID:31101820

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AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated hippocampal degeneration

Amado DA, Rieders JM, Diatta F, Hernandez-Con P, Singer A, Zhang J, Lancaster E, Davidson BL, Chen-Plotkin AS, Mol Ther 27(2): , 2019

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.

Maguire JA, Gagne AL, Gonzalez-Alegre P, Davidson BL, Shakkottai V, Gadue P, French DL., Stem Cell Res 34(): 101361, 2019, PMID:30611021

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CRISPPR to the rescue: advances in gene editing for the FMR1 gene.

Yrigollen CM, Davidson BL, Brain Sci 9(1): pii:E17, 2019, PMID:30669625

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Viral vectors for gene transfer

Chen YH, Keiser MS, Davidson BL, Curr Protoc Mouse Biol, 2018, PMID:30485696

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