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

Scott Harper, PhD

Scott  Harper, PhD

Center for Gene Therapy
Principal Investigator

Neurology
Principal Investigator

Neurology Residency
Faculty

Neuromuscular Disorders
Principal Investigator

Neurosciences
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

Biography

View CV »

Gender:

  • 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

2013–present

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

Publications

  • 2014. RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice.  MOLECULAR THERAPY-NUCLEIC ACIDS. Vol. 3, no. April 1.
  • 2013. Dose-Dependent Toxicity of Humanized Renilla Reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle.  MOLECULAR THERAPY. Vol. 21, no. June 1.
  • 2013. Dose-dependent Toxicity of Humanized Renilla reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle.  MOLECULAR THERAPY-NUCLEIC ACIDS. Vol. 2, no. April 1.
  • LM Wallace, A Moreo, KR Clark, and SQ Harper. 2013. LM Wallace, A Moreo, KR ClarDose-dependent toxicity of humanized Renilla reniformis GFP limts its utility as a reporter gene in mouse muscle.  Molecular Therapy Nucleic Acids. Vol. 2, no. April 1: e86-e86.
  • 2013. Molecular dissection of dystrophin identifies the docking site for nNOS.  PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. Vol. 110, no. 2. (January 8).
  • SQ Harper. 2013. Molecular dissection of dystrophin identifies the docking site for nNOS.  PNAS. Vol. 110(2), no. January: 387-8.
  • SQ Harper. 2013. Molecular dissection of dystrophin identifies the docking site for nNOS.  PNAS. Vol. 110(2), no. January 1: 387-8-387-8.
  • 2012. RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies.  CURRENT GENE THERAPY. Vol. 12, no. 4. (August 1).
  • 2012. Conditional over-expression of PITX1 causes skeletal muscle dystrophy in mice.  BIOLOGY OPEN. Vol. 1, no. 7. (July 15).
  • 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 1): 1417-23-1417-23.
  • 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.
  • 2012. RNA Interference Inhibits DUX4-Induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy.  MOLECULAR THERAPY. Vol. 20, no. May 1.
  • 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.
  • 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 1: 629-639-629-639.
  • 2011. Construction of permanently inducible miRNA-based expression vectors using site-specific recombinases.  BMC BIOTECHNOLOGY. Vol. 11, no. November 16.
  • 2011. RNA Interference Improves Myopathic Phenotypes in Mice Over-expressing FSHD Region Gene 1 (FRG1).  MOLECULAR THERAPY. Vol. 19, no. 11. (November 1).
  • 2011. DUX4, a Candidate Gene for Facioscapulohumeral Muscular Dystrophy, Causes p53-Dependent Myopathy In Vivo.  ANNALS OF NEUROLOGY. Vol. 69, no. 3. (March 1).
  • RL Boudreau, SE Garwick-Coppens, J Liu, LM Wallace,and SQ Harper. 2011. Rapid Cloning and Validation of MicroRNA Shuttle Vectors: A Practical Guide. In RNA Interference Methods.
  • 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.
  • 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.
  • SQ Harper. 2011. Neuromethods Introduction. In RNA Interference Methods.
  • 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.
  • Garwick-Coppens, SE and Harper SQ. 2011. Construction of permanently inducible miRNA expression vectors using site-specific recombinases.  BMC Biology.
  • 2010. PP2A:B56 epsilon, a Substrate of Caspase-3, Regulates p53-dependent and p53-independent Apoptosis during Development.  JOURNAL OF BIOLOGICAL CHEMISTRY. Vol. 285, no. 45. (November 5).
  • 2010. Treatment response and tolerability of frovatriptan in patients reporting short- or long-duration migraines at baseline.  CURRENT MEDICAL RESEARCH AND OPINION. Vol. 26, no. 9. (September 1).
  • 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.
  • 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.
  • L.M. Wallace, S.E. Garwick, and S.Q. Harper. 2010. RNAi therapy for dominant muscular dystrophies and other myopathies. In Muscle Gene Therapy. Edited by Dongsheng Duan.
  • 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.
  • 2009. Progress and Challenges in RNA Interference Therapy for Huntington Disease (vol 66, pg 933, 2009).  ARCHIVES OF NEUROLOGY. Vol. 66, no. 10. (October 1).
  • 2009. Progress and Challenges in RNA Interference Therapy for Huntington Disease.  ARCHIVES OF NEUROLOGY. Vol. 66, no. 8. (August 1).
  • 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
  • SQ Harper. 2009. Progress and challenges in RNAi therapy for Hunington's Disease.  Archives of Neurology. Vol. 66, no. 8. (January 1): 933-938-933-938.
  • 2008. The Bifunctional microRNA miR-9/miR-9*Regulates REST and CoREST and Is Downregulated in Huntington's Disease.  JOURNAL OF NEUROSCIENCE. Vol. 28, no. 53. (December 31).
  • 2008. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.  Proceedings of the National Academy of Sciences of the United States of America. Vol. 105, no. 15. (April 8).
  • 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
  • 2008. Lentivirus-mediated RNA interference in mammalian neurons.
  • 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
  • SQ Harper, P Gonzalez-Alegre. 2008. Lentivirus-mediated RNA interference in mammalian neurons.  Methods Mol Biol.. Vol. 442, no. January 1: 95-112-95-112.
  • 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
  • 2006. Optimization of Feline Immunodeficiency Viral Vectors for RNA Interference.  MOLECULAR THERAPY. Vol. 13, no. May 1.
  • P Allaire, B Ritter, S Thomas, J Burman, A Denisov, V Legendre-Guillmen, SQ Harper, B.L. Davidson, K. Gehring, P. McPherson. 2006. Connecdenn, A DENN domain-containing protein functioning in synaptic vesicle endocytosis.  Journal of Neuroscience. Vol. 26, no. 51. (January 1): 13202-13212-13202-13212.
  • 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
  • 2005. CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo.  JOURNAL OF NEUROSCIENCE. Vol. 25, no. 40. (October 5).
  • 2005. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.  PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. Vol. 102, no. 16. (April 19).
  • 2005. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury.  MOLECULAR THERAPY. Vol. 11, no. 2. (February 1).
  • B.L. Davidson and S.Q. Harper.  Viral delivery of short hairpin RNAs. (2005) Methods in Enzymology 392, 145-173. PubMed ID: 15644180
  • BL Davidson, SQ Harper. 2005. Viral delivery of short hairpin RNAs.  Methods in Enzymology. Vol. 392, no. January 1: 145-173-145-173.
  • SQ Harper, and BL Davidson. 2005. Plasmid-based RNA interference.  Methods in Molecular Biology. Vol. 309, no. January 1: 219-235-219-235.
  • 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, P.D. Staber, X. He, S.L. Eliason, I.H. Martins, L. Yang, H.L. Paulson, R.M. Kotin, B.L. Davidson. 2005. RNA interference improves behavioral and neuropathological abnormalities in a mouse model for Huntington's disease.  Proceedings of the National Academy of Sciences, USA. Vol. 102, no. 16. (January 1): 5820-5825-5820-5825.
  • S.Q. Harper and B.L. Davidson.  Plasmid-based RNA interference.  (2005) Methods in Molecular Biology 309, 219-235. PubMed ID: 15990403
  • 2005. Plasmid-based RNA interference: construction of small-hairpin RNA expression vectors.  Methods in molecular biology (Clifton, N.J.). Vol. 309, no. January 1.
  • 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
  • 2004. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6.  MOLECULAR THERAPY. Vol. 10, no. 4. (October 1).
  • 2004. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.  NATURE MEDICINE. Vol. 10, no. 8. (August 1).
  • 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
  • 2003. Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart.  CIRCULATION. Vol. 108, no. 13. (September 30).
  • 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
  • 2002. Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin.  NEUROMUSCULAR DISORDERS. Vol. 12, no. October 1.
  • 2002. Spectrin-like repeats from dystrophin and alpha-actinin-2 are not functionally interchangeable.  HUMAN MOLECULAR GENETICS. Vol. 11, no. 16. (August 1).
  • SQ Harper, M Hauser, C DelloRusso, D Duan, RW Crawford, S Phelps, HA Harper, AS Robinson, JF Engelhardt, SV Brooks, JS Chamberlain. 2002. Modular flexability of dystrophin: Implications for gene therapy of DMD.  Nature Medicine. Vol. 8, no. 3. (January 1): 253-261-253-261.
  • 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
  • 1998. Phase behavior of polystyrene/polybutadiene and polystyrene/hydrogenated polybutadiene mixtures: Effect of the microstructure of polybutadiene.  MACROMOLECULES. Vol. 31, no. 2. (January 27).
  • 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.
  • KM Flanigan and SQ Harper. Facioscapulohumeral Muscular Dystrophy. In Muscle Disease: Pathology and Genetics. Edited by Goebel, Sewry, and Weller.
  • 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.
  • 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.
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