Scott Harper, Ph.D. :: Nationwide Children's Hospital, Columbus, Ohio

Scott Harper, PhD

Scott  Harper, PhD

Center for Gene Therapy
Principal Investigator

Principal Investigator

Neurology Residency

Neuromuscular Disorders
Principal Investigator

Principal Investigator

Contact Information

The Research Institute at Nationwide Children's Hospital
700 Children's Drive
Columbus, Ohio 43205 [ map ]
PH: (614) 355.2893
FX: (614) 355.5247
Email Me


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  • Male

Languages Spoken:

  • English

Research Interests

Research Center:

Areas of Interest:

  • Dr. Harper's laboratory studies gene therapy for dominant genetic diseases using RNA interference (RNAi), with particular focus on muscular dystrophy and neurodegenerative disease. The Harper lab utilizes a broad range of tools, including molecular biology, biochemistry, RNAi, gene therapy vectors, and animal models for muscular dystrophy and peripheral neuropathy.

Education and Training

Undergraduate School

  • Saginaw Valley State University
    Date Completed: 06/30/1996

Post Doctoral

  • University of Michigan Medical School
    Date Completed: 06/30/2002

Post Doctoral

  • University of Iowa College of Medicine
    Date Completed: 06/30/2007

Professional Experience


  • Associate Professor, Department of Pediatrics, The Ohio State University College of Medicine


  • SQ Harper. 2013. Molecular dissection of dystrophin identifies the docking site for nNOS.  PNAS. Vol. 110(2), no. January: 387-8.
  • LM Wallace, J Liu, JS Domire, SE Garwick-Coppens, SE Guckes, JR Mendell, KM Flanigan, and SQ Harper. 2012. RNA Interference inhibits DUX4-induced muscle toxicity in vivo: Implications for a targeted FSHD therapy.  Molecular Therapy. Vol. 20, no. 7. (July): 1417-23.
  • SN Pandy, J Cabotage, R Shi, M Dixit, M Sutherland, J Liu, S Muger, SQ Harper, K Nagaraju, and YW Chen. 2012. Conditional over-expression of PIXT1 causes skeletal muscle atrophy in mice.  Biology Open. Vol. 1, no. January: 629-639.
  • Boudreau, RL, Garwick-Coppens, SE, Liu, J, Wallace, LM, and Harper, SQ. 2011. Rapid Cloning and Validation of MicroRNA Shuttle Vectors: A Practical Guide. In RNA Interference Methods. New York: Springer Science+Business Media, LLC.
  • Garwick-Coppens, SE and Harper SQ. 2011. Construction of permanently inducible miRNA expression vectors using site-specific recombinases.  BMC Biology.
  • Wallace, LM, Garwick-Coppens, SE, Tupler, R, and Harper, SQ. 2011. RNA Interference Improves Myopathic Phenotypes in Mice Over-Expressing FSHD Region Gene 1 (FRG1).  Molecular Therapy.
  • Jin Z, Wallace L, Harper SQ, Yang J. 2010. PP2A:B56{epsilon} a substrate of caspase-3 regulates p53-dependent and p53-independent apoptosis during development.  J Biol Chem. Vol. 285, no. 45. (January): 34493-34502.
  • Wallace LM, Garwick SE, Mei W, Belayew A, Coppee F, Ladner KJ, Guttridge D, Yang J, Harper SQ. 2010. DUX4 a candidate gene for facioscapulohumeral muscular dystrophy causes p53-dependent myopathy in vivo.  Ann Neurol.
  • Kelman L, Harper SQ, Hu X, Campbell JC. 2010. Treatment response and tolerability of frovatriptan in patients reporting short-or-long duration migraines at baseline.  Curr Med Res Opin. Vol. 26, no. 9. (January): 2097-2104.
  • Harper,S,Q. 2009. Progress and Challenges in RNA Interference Therapy for Huntington Disease (vol 66, pg 933, 2009).  ARCHIVES OF NEUROLOGY. Vol. 66, no. 10. (October): 1272-1272.
  • S.Q. Harper. Progress and challenges in RNAi therapy for Huntington's Disease. Arch Neurol. 2009 Aug;66(8):933-8. Review. Erratum in: Arch Neurol. 2009 Oct;66(10):1272. PubMed ID: 19667213
  • JL McBride, RL Boudreau, SQ Harper (SHARED FIRST AUTHORSHIP), PD Staber, AM Monteys, I Martins, BL Gilmore, H Burstein, RW Peluso, B Polisky, BJ Carter, BL Davidson. 2008. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.  Proc Natl Acad Sci U S A. Vol. 105, no. 15. (April): 5868-5873.
  • S.Q. Harper and Gonzalez-Alegre P. Lentivirus-mediated RNA interference in mammalian neurons. (2008) Methods in Molecular Biology 442, 95-112. PubMed ID: 18369781
  • A. Packer, Y. Xing, S.Q. Harper, L. Jones, and B.L. Davidson. The bi-functional microRNA mir9/mir9* regulates REST and coREST and is down-regulated in Huntington's desease (2008) Journal of Neuroscience, 28(53):14341-6 PubMed ID: 19118166
  • J.L. McBride*, R.L. Boudreau*, S.Q. Harper* (shared first authorship), A. Mas Monteys, P.D. Staber, I. Martins, B. Gilmore, H. Burstein, R.W. Peluso, B. Polisky, B.J. Carter, and B.L. Davidson. MicroRNA shuttles mitigate short-hairpin RNA mediated toxicity in the brain: Implications for therapeutic development of RNA interference. (2008) Proceedings of the National Academy of Sciences, USA 105(15): 5868-73. PubMed ID: 18398004
  • S.Q. Harper, P.D. Staber, C.R. Beck, S.K. Fineberg, C.S. Stein, D. Ochoa, and B.L. Davidson.  Optimization of feline immunodeficiency viral vectors for RNA interference.  (2006), Journal of Virology 80(19), 9371-9380 PubMed ID: 16973543
  • P. Allaire, B. Ritter, S. Thomas, J. Burman, A. Denisov, V. Legendre-Guillmen, S.Q. Harper, B.L. Davidson, K. Gehring, and P. McPherson. Connecdenn, A DENN domain-containing protein functioning in synaptic vescle endocytosis. (2006) Journal of Neuroscience 26(51) 13202-13212 PubMed ID: 17182770
  • B.L. Davidson and S.Q. Harper.  Viral delivery of short hairpin RNAs. (2005) Methods in Enzymology 392, 145-173. PubMed ID: 15644180
  • M. Liu, Y. Yue, S.Q. Harper, R.W. Grange, J.S. Chamberlain, and D. Duan.  Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. (2005) Molecular Therapy 11(2), 245-256. PubMed ID: 15668136
  • S.Q. Harper and B.L. Davidson.  Plasmid-based RNA interference.  (2005) Methods in Molecular Biology 309, 219-235. PubMed ID: 15990403
  • S.Q. Harper, P.D. Staber, X. He, S.L. Eliason, I.H. Martins, L. Yang, H.L. Paulson, R.M. Kotin, and B.L. Davidson.  RNA interference improves behavioral and neuropathological abnormalities in a mouse model for Huntington’s disease. (2005) Proceedings of the National Academy of Sciences, USA.  102(16), 5820-5825. PubMed ID: 15811941
  • M.J. Blankinship, P. Gregorovic, J.M. Allen, S.Q. Harper, H.A. Harper, C. Halbert, A.D. Miller, and J.S. Chamberlain.  Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype-6. (2004) Molecular Therapy 10(4), 671-678. PubMed ID: 15451451
  • H. Xia, Q. Mao, S.L. Eliason, S.Q. Harper, I.H. Martins, H.T. Orr, H.L. Paulson, L. Yang, R.M. Kotin, and B.L. Davidson.  RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. (2004) Nature Medicine 10(8), 816-820. PubMed ID: 15235598
  • Y. Yue, Z. Li, S.Q. Harper, R.L. Davisson, J.S. Chamberlain, and D. Duan. Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. (2003) Circulation 108(13), 1626-1632. PubMed ID: 12952841
  • S.Q. Harper, R.W. Crawford, C. DelloRusso, and J.S. Chamberlain.  Spectrin-like repeats from dystrophin and ?-actinin are not functionally interchangeable.  (2002) Human Molecular Genetics, 11(16), 1807-1815. PubMed ID: 12140183
  • S.Q. Harper, M. Hauser, C.  DelloRusso, D. Duan, R.W. Crawford, S. Phelps, H.A. Harper, A.S. Robinson, J.F. Engelhardt, S.V. Brooks, and J.S. Chamberlain.  (2002) Modular flexibility of dystrophin:  Implications for gene therapy of DMD.  Nature Medicine, 8(3), 253-261. PubMed ID: 11875496
  • LM Wallace, A Moreo, KR Clark, and SQ Harper. Dose dependent toxicity of humanized Renilla reniformis GFP limits its utility as a reporter gene in mouse muscle.  Molecular Therapy Nucleic Acids.
  • Harper, SQ. Molecular dissection of dystrophin identifies the docking site for nNOS.  PNAS.
  • LM Wallace, A Moreo, KR Clark, and SQ Harper. Dose dependent toxicity of humanized Renilla Reniformis GFP (hrGFP) limits utility as a reporter gene in mouse muscles.  Molecular Therapy Nucleic Acids.
  • E Ansseau, SE Garwick-Coppens, JS Domire, LM Wallace, A Belayew, and SQ Harper. V5 epitope tag causes aberrant alternative splicing: a cautionary case study.  BMC Molecular Biology.
Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000