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.


BS (Biology Major/Chemistry Minor; High Distinction), Nebraska Wesleyan University, 1981
PhD (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, 1977-81
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
Carver Research Program of Excellence, University of Iowa, 2007-14
Regents Faculty Excellence Award, 2007
Eureka Award, National Institutes of Health, 2008
Mathilde Solowey Award, National Institutes of Health, 2008
Distinguished Graduate Lecture, University of Michigan Dept of Biological Chemistry, 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
Leslie Gehry Prize for Innovation in Science, Hereditary Disease Foundation, 2015
Member, American Academy of Arts and Sciences, 2017
John B. Penney, Jr. MD, Memorial Lecture, Mass General Hospital, 2017
Member, International Selection Committee, 2018 Bower Award for Achievement in Science, The Franklin Institute, 2017-18
FE Bennett Memorial Lectureship Award, American Neurological Association, 2018
Member, The College of Physicians of Philadelphia, 2018
Member, National Academy of Medicine
Reviewer, National Academy of Medicine Emerging Leaders in Health and Medicine (ELHM) Scholars, 2022-, 2019
President, American Society of Gene and Cell Therapy, 2021-22
Katherine A. High Chair in Cell and Gene Therapy, 2022
Safadi Lecturer, Safadi Program of Excellence in Clinical and Translational Neuroscience, University of Chicago, 2022
Dr. John W. Schut Research Achievement Award, National Ataxia Foundation, 2023

Memberships and Professional Organizations

NIH/NICHD, 1995 - 1999
American Association for the Advancement of Science (AAAS), 1995 - Present
NIH Study Sections, 1995 - Present
National Gene Vector Laboratory, 1996 - 2007
American Society for Gene and Cell Therapy, 1997 - Present
Hunters Hope, 2000 - 2002
Batten Disease Support & Research Association (BDSRA), 2000 - Present
Amytrophic Lateral Sclerosis Association, 2000 - 2003
Oxford Biomedica Inc, 2001 - 2006
TIGEM, 2002 - 2002
Sirna Therapeutics, 2004 - 2007
TIGEM, 2006 - 2006
Hereditary Disease Foundation, 2006 - 2009
Sirna Therapeutics, 2006 - 2008
Centre de Genomica Regulacio, Barcelona Spain, 2007 - Present
TIGEM, 2007 - 2007
Comitato Telethon Fondazione, 2007 - 2010
Robert Packard Center for ALS Research at Johns Hopkins, 2007 - Present
Oregon National Primate Research Center, 2008 - Present
Wellstone Muscular Dystrophy Center, 2008 - 2011
TIGEM, 2008 - 2008
NCL Foundation, Hamburg, Germany, 2008 - Present
UMass, 2009 - Present
TIGEM, Naples Italy, 2009 - Present
Basal Ganglia Disorders Linnaeus Consortium, Lund, Sweden, 2009 - 2014
MDRNA, 2009 - 2010
Comitato Telethon Fondazione, 2010 - 2011
Marina Biotech, 2010 - 2012
TIGEM, 2011 - 2011
NIH, 2011 - 2016
Hereditary Disease Foundation, 2012 - 2015
nLIFE, 2012 - Present
NIH Common Fund, 2013 - Present
Spark Therapeutics Inc., 2014 - 2019
Washington University School of Medicine, St. Louis, MO, 2014 - 2015
NIH Common Fund, 2014 - Present
TIGEM, 2014 - 2014
NIH/NINDS, 2014 - Present
NIH/NINDS, 2014 - 2014
American Society of Human Genetics, 2015 - Present
National Ataxia Foundation, 2015 - Present
Huntington Study Group, 2015 - 2018
European Commission, 2015 - Present
Intrathecal Administration of scAAV9/Jet-GAN for the Treatment of Giant Axonal Neuropathy (NIH Sponsored), 2015 - Present
Sarepta Therapeutics, Cambridge, A, 2015 - 2020
NIH/NINDS, 2016 - 2016
Intellia Therapeutics, 2016 - 2020
NIH/NINDS, 2016 - 2016
Friends of Telethon, Italia, 2016 - Present
Elected to American Academy of Arts and Sciences, 2017 - Present
Johns Hopkins University, 2017 - 2020
STXBP1 Foundation, 2017 - Present
Anhui IsoTex (Yun Tai) Biotech, Inc., 2017 - 2018
Franklin Institute Awards, 2017 - 2018
Prevail Therapeutics, 2018 - 2020
Spirovant, 2018 - Present
The College of Physicians of Philadelphia,, 2018 - Present
American Society of Human Genetics, 2018 - Present
Homology Medicines, Inc., 2019 - Present
Panorama Medicine, Inc., 2019 - Present
Pfizer, 2019 - 2020
Elected to National Academy of Medicine, 2019 - Present
Patch Bio, 2020 - Present
Moment Bio, 2020 - Present
Voyager Therapeutics, 2020 - Present
Saliogen Therapeutics, 2020 - Present
National Academy of Sciences, 2022 - Present
Fondazione Telethon, 2022 - 2024
Latus Biosciences, 2022 - Present

Web Links

Selected Publications

VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF

Carrell EM, Chen YH, Ranum PT, Coffin SL, Singh LN, Tecedor L, Keiser MG, Hudry E, Hyman BT, Davidson BL, Mol Ther Nucleic Acids 33(): 296-304, 2023, PMID:37547292

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Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching

Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Maguire JA, Gagne AL, Heller EA, French DL, Davidson, BL, Prosser, BL, Nat Commun 14(1): 2628, 2023, PMID:37149717

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Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain

Simpson, BP, Yrigollen CM, Izea A, Davidson BL, Mol Ther 31(3): 760-773, 2023, PMID:36617193

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Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates

Ranum PT, Tecedor L, Keiser MS, Chen YH, Leib DE, Liu X, Davidson, BL, Mol Ther 31(3): 609-612, 2023, PMID:36610400

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Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing

Fang L, Monteys AM, Durr A, Keiser M, Cheng C, Harapanahalli A, Gonzalez-Alegre P, Davidson BL, Wang K, HGG Adv 4(1): 100146, 2022, PMID:36262216

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Huntington's disease phenotypes are improved via mTORC1 modulation by small molecule therapy

St-Cyr S, Child DD, Giaime E, Smith AR, Pascua CJ, Hahm S, Saiah E, Davidson BL, PLoS One 17(8): e0273710, 2022, PMID:36037192

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J (American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration), Mol Ther 30(8): 2646-2663, 2022, PMID:35690906

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Mapping PTBP splicing in human brain identifies targets for therapeutic splice switching SNYGAP1

Dawicki-McKenna JM, Felix AJ, Waxman EA, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Heller EA, French DL, Davidson BL, Prosser BL, bioRxiv, 2022

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bonnemann C, Buxbaum JD, Corcoran G, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CF, Sahin M, (Gene Therapy Workshop Faculty), Mol Ther 30(7): 2416-2428, 2022, PMID:35585789

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The molecular and behavioral phenotypes altered in CGG knock-in mice edited using CRISPR

Yrigollen CM, Simpson B, Lim E, Chen YH, Davidson B, Presented at the 25th annual meeting of the American Society for Gene and Cell Therapy, Washington, DC, 2022