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
Distinguished Graduate Lecture, University of Michigan Dept of
Biological Chemistry, Ann Arbor, MI, 2011
SJ DeArmond Lecture, American Association of Neuropathologists, 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

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

Web Links

Selected Publications

Mis-splicing in Huntington's disease: harnessing the power of comparative transcriptomics

Xing Y, Wang R, Davidson BL, Trends Neurosci S0166-2236(21): 00209-5, 2021 (in press)

Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain

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, Nat Med, 2021 (in press), PMID:34663988

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Design and validation of a multi-point injection technology for MR-guided convection enhanced delivery in the brain

Prezelski K, Keiser M, Stein J, Lucas TH, Davidson B, Gonzalez P, Vitale F, Front Med Technol, 2021 (in press)

Next Generation Strategies for Gene-Targeted Therapies of Central Nervous System Disorders: A Workshop Summary

Morris JA, Boshoff CH, Schor NF, Wong LM, Gao G, Davidson BL, Mol Ther, 2021 (in press), PMID:34547463

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Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics

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, Hum Mol Genet, 2021 (in press), PMID:34296279

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An orally available, brain penetrant, small molecule lowers Huntingtin levels by enhancing pseudoexon inclusion

Keller CG, Shin Y, Monteys AM, Renaud N, Beibel M, Teider N, Peters T, Faller T, St-cyr S, Knehr J, Roma G, Quiroz AR, HIld M, Lukashev D, Theil D, Dales N, Cha J-H, Borowsky B, Dolmetsch R, Davidson BL*, Sivasankaran R* (*authors contributed equally), Nat Commun, 2021 (in press)

Gene therapy for ALS: A review

Amado DA, Davidson BL, Mol Ther, 2021, PMID:33839324

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Regulated control of gene therapies by drug-induced splicing

Monteys AM, Hundley AA, Ranum PT, Tecedor L, Muehlmatt A, Lim E, Lukashev, Sivasankaran R, Davidson BL, Nature 596(7871): , 2021, PMID:34321659

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Mavis Agbandje-McKenna's Lifelong Commitment to Teaching and Research

Byrne BJ, McCall AL, Davidson BL, Samulski RJ, Hum Gene Ther 32(7-8): 319-320, 2021, PMID:33891506

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Deubiquitinase USP7 contributes to the pathogenicity of spinal and bulbar muscular atrophy

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, J Clin Invest 131(1): :e134565, 2021, PMID:33170804

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