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
Director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics
Katherine A High Presidential Scholar Endowed Chair in Cell and Gene Therapy
Children’s Hospital of Philadelphia (CHOP)
Contact InformationChildren’s Hospital of Philadelphia
Department of Pathology and Laboratory Medicine
Colket Translational Research Building, Rm. 5060
3501 Civic Center Boulevard
Philadelphia, PA 19104
Tel: 267-426-0929
Fax: 215-590-3660
Email: davidsonbl@chop.edu
Research Expertise
Keywords:
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:
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.
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- Recessive, childhood onset neurodegenerative disease, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease;
- Dominant genetic diseases, specifically the CAG repeat disorders, Huntington’s disease and spinal cerebellar ataxia;
- Understanding how changes in the transcriptome impact neural development and neurodegenerative disease processes.
Graduate Groups
Cell and Molecular Biology
Neuroscience
Education
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, 1982-83
Postdoctoral Fellow, University of Michigan, 1988
Research Investigator, University of Michigan, 1990-92
Assistant Research Scientist, University of Michigan, 1992-94
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
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
American Association for the Advancement of Science (AAAS), 1995-present
-------- Electorate Nominating Committee, Medical Sciences, 2009-12
-------- Medical Sciences, Chair, 2012
-------- Electorate Nominating Committee, Neuroscience, 2019-22
NIH
-------- Study Sections, Ad Hoc Reviewer, 1995-present
-------- NIH/NICHD Mental Retardation Research Committee, 1995-99
-------- TAGS Study Section, 2011-16
-------- Common Fund, Expert Scientific Panel, 2013-present
-------- Common Fund, Transformative R01 Editor Panel, Co-Chair, 2014-present
-------- NIH/NINDS, Nat’l Advisory Neurological Disorders & Stroke Council, 2014-present
-------- NIH/NINDS, Blue Ribbon Panel for Review of Intramural Programs, 2014
-------- NIH/NINDS, Working Group of Council of Diversity, 2016
-------- NIH/NINDS, Scientific Director Search Committee, 2016
National Gene Vector Laboratory, Scientific Review Board, 1996-2007
American Society for Gene and Cell Therapy, 1997-present
-------- Neuro Committee, 1999-2007 (Chair 1999-2002)
-------- Board of Directors, 2000-03
-------- Education and Nominating Committees, 2003-06
-------- Treasurer, 2005-11
-------- Chair, Advisory Committee, 2016-present
-------- Program Committee, 2018-present
-------- Vice President-Elect, 2019
-------- Vice President, 2020
-------- President, 2021-22
-------- Annual Meeting Program Committee, 2021
Amytrophic Lateral Sclerosis Association, Study Section, 2000-03
Batten Disease Support & Research Association, Scientific Advisory Board, 2000-present
Hunters Hope, Scientific Advisory Board, 2000-02
Oxford Biomedica Inc., Scientific Advisory Board, 2001-06
Telethon Institute of Genetics and Medicine (TIGEM)
-------- Site Visit Team, Milan, Italy, 2002, 2006, 2007, 2008, 2009, 2011, 2014
-------- Search Committee for New Director, 2023-present
Sirna Therapeutics
-------- Consultant, 2004-07
-------- Scientific Advisory Board, 2006-08
Hereditary Disease Foundation, Scientific Advisory Board, 2006-09, 2012-15
Centre de Genomica Regulacio, Barcelona Spain, Reviewer, 2007-present
Comitato Telethon Fondazione
-------- Scientific Advisory Board, 2007-10
-------- Scientific Committee, Vice Chair, 2010-11
Robert Packard Center for ALS Research at Johns Hopkins, Scientific Advisory Board, 2007-present
NCL Foundation, Scientific Advisory Board, Hamburg, Germany, 2008-present
Oregon National Primate Research Center, Scientific Advisory Board, 2008-present
Wellstone Muscular Dystrophy Center, Scientific Advisory Board, 2008-11
Basal Ganglia Disorders Linnaeus Consortium, Scientific Advisory Board, 2009-14
MDRNA, Scientific Advisory Board, 2009-10
UMass, CNS Therapy Advisory Board, 2009-present
Marina Biotech, Scientific Advisory Board, 2010-12
nLIFE, Scientific Advisory Board, 2012-present
Cell & Molecular Biology (CAMB) Graduate Group, Univ of Pennsylvania, 2014-present
Mahoney Institute for Neurosciences, University of Pennsylvania, 2014-present
-------- Neuroscience Advisory Committee, 2017-present
Neuroscience Graduate Group, University of Pennsylvania, 2014-present
Spark Therapeutics Inc., Co-Founder, 2014-19
Washington University School of Medicine, External Advisory Committee, Pathophysiology and Global Therapy for Krabbe Disease, 2014-15
American Society of Human Genetics, Social Issues Committee, 2015-present
European Commission, Advisory Board, 2015-present
Huntington Study Group, Scientific Advisory Committee, 2015-18
Intrathecal Administration of scAAV9/Jet-GAN for the Treatment of Giant Axonal Neuropathy (NIH Sponsored), Data Safety Monitoring Board, 2015-present
National Ataxia Foundation, Medical Research Advisory Board (MRAB), 2015-present
Research Advisory Council, Children’s Hospital of Philadelphia, 2015-present
Sarepta Therapeutics, Cambridge, A, Scientific Advisory Board, 2015-20
Dean’s Advisory Council, Perelman School of Medicine at the University of Pennsylvania, 2016-present
Friends of Telethon, Scientific Advisory Board, 2016-present
Institutional T32 and K12 Clinical Scholars Program, Internal Advisory Committee, Children’s Hospital of Philadelphia, 2016-present
Intellia Therapeutics, Scientific Advisory Board, 2016-20
Research Information Services Advisory Committee (RISAC), Children’s Hospital of Philadelphia, 2016-present
Research Space Committee, Children’s Hospital of Philadelphia, Chair, 2016-present
American Academy of Arts and Sciences, 2017-present
Anhui IsoTex (Yun Tai) Biotech, Inc., Scientific Advisory Board, 2017-18
Franklin Institute Awards, International Selection Committee, 2018 Bower Award & Prize for Achievement in Science, 2017-18
Johns Hopkins University, Packard Center Board of Advisors, 2017-20
Penn Medicine Standing Chairs and Directors of Centers, 2017-present
Penn Translational Neuroscience Center, Scientific Advisory Board, 2017-present
STXBP1 Foundation, Scientific Advisory Board, 2017-present
American Society of Human Genetics, 2018-present
College of Physicians of Philadelphia, 2018-present
Prevail Therapeutics, Scientific Advisory Board, 2018-20
Spirovant, Founder, 2018-present
Homology Medicines, Inc., Scientific Advisory Board, 2019-present (Chair 2021-present)
National Academy of Medicine, 2019-present
-------- Emerging Leaders in Health and Medicine (ELHM) Scholars, Reviewer, 2022-present
Panorama Medicine, Inc., Scientific Advisory Board, 2019-present
Pfizer, TASAP, 2019-20
Children’s Hospital of Philadelphia, Bridge Funding Committee, Chair, 2020-present
Moment Bio, Scientific Advisory Board, 2020-present
Patch Bio, Scientific Advisory Board, 2020-present
Saliogen Therapeutics, Scientific Advisory Board, 2020-present
Voyager Therapeutics, Scientific Advisory Board, 2020-present
Research Institute Summer Scholars Program (CRISSP) R25, Executive Committee, Children’s Hospital of Philadelphia, 2021-present
Fondazione Telethon, Scientific Committee, Panel Chair, 2022-24
Latus Biosciences, Founder, 2022-present
National Academy of Sciences, Forum on Neuroscience and Nervous System Disorders, 2022-present
Senate Committee on Academic Freedom and Responsibility (CAFR), University of Pennsylvania, 2022-24
Bioengineering Graduate Group, Penn Engineering, 2023-present
Lifespan Brain Institute (LiBi), Internal Advisory Board, University of Pennsylvania and Children’s Hospital of Philadelphia, 2023-present
Web Links
Selected Publications
A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency
Pham V, Sertori Finoti L, Cassidy MM, Maguire JA, Gagne AL, Waxman EA, French DL, King K, Zhou Z, Gelb MH, Wongkittichote P, Hong X, Schlotawa L, Davidson BL, Ahrens-Nicklas RC. Mol Genet Metab 141(2):108116, Feb 2024. doi: 10.1016/j.ymgme.2023.108116. Epub 2023 Dec 21. PMID: 38161139.
AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS
Amado DA, Robbins AB, Smith AR, Whiteman KR, Chillon Bosch G, Chen Y, Fuller JA, Izda A, Nelson S, Dichter AI, Monteys AM, Davidson BL. bioRxiv [Preprint]. 2024 Feb 2:2024.01.31.578314. doi: 10.1101/2024.01.31.578314. PMID: 38352376; PMCID: PMC10862885.
Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability
Gall-Duncan T, Luo J, Jurkovic C-M, Fischer LA, Fujita K, Deshmukh AL, Harding RJ, Tran S, Mehkary M, Li V, Leib DE, Chen R, Tanaka H, Mason AG, Levesque D, Khan M, Razzaghi M, Prasolava T, Lanni S, Sato N, Caron M-C, Panigrahi GB, Wang P, Lau R, Castel AL, Masson J-Y, Tippet L, Turner C, Spies M, LaSpada AR, Campos EI, Curtis MA, Boisvert F-M, Faull RLM, Davidson BL, Nakamori M, Okazawa H, Wold MS, Pearson CE. Cell 186(22): 4898-4919.e25, Oct 2023. doi: 10.1016/j.cell.2023.09.008. Epub 2023 Oct 11. PMID: 37827155.
APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
Jackson RJ, Keiser MS, Meltzer JC, Fykstra DP, Dierksmeier SE, Melloni A, Nakajima T, Tecedor L, Ranum PT, Carrell E, Chen YH, Holtman DM, Davidson BL, Hyman BT. bioRxiv [Preprint]. 2023 Aug 16:2023.08.14.552850. doi: 10.1101/2023.08.14.552850. PMID: 37645718; PMCID: PMC10461997.
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, Jul 2023. doi: 10.1016/j.omtn.2023.07.016. PMID: 37547292; PMCID: PMC10400871.
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, May 2023. doi: 10.1038/s41467-023-38273-3. PMID: 37149717; PMCID: PMC10164156.
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain
Simpson BP, Yrigollen CM, Izda A, Davidson BL. Mol Ther 31(3):760-773, Mar 2023. doi: 10.1016/j.ymthe.2023.01.004. Epub 2023 Jan 6. PMID: 36617193; PMCID: PMC10014281.
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, Mar 2023. doi: 10.1016/j.ymthe.2022.12.018. Epub 2023 Jan 6. PMID: 36610400; PMCID: PMC10014218.
Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing
Fang L, Monteys AM, Dürr A, Keiser M, Cheng C, Harapanahalli A, Gonzalez-Alegre P, Davidson BL, Wang K. HGG Adv 4(1):100146, Sep 2022. doi: 10.1016/j.xhgg.2022.100146. Erratum in: HGG Adv. 2023 Jun 20;4(3):100212. PMID: 36262216; PMCID: PMC9574884.
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, Aug 2022. doi: 10.1371/journal.pone.0273710. PMID: 36037192; PMCID: PMC9423655.
