Scott Q. Harper, Ph.D.
The Ohio State University School of Medicine
Department of Pediatrics
Nationwide Children’s Research Institute
700 Children’s Drive, Rm. WA3015
Columbus, OH 43205
Phone: (614) 355-2893
Education & Training:
Saginaw Valley State University, Saginaw, MI, 1996 BS in Biology
University of Michigan Medical School, 2002, Ph.D. in Cellular and Molecular Biology
University of Iowa College of Medicine, Iowa City, 2007, Postdoctoral Fellow
The Harper Lab is focused on developing RNAi-based treatments for dominantly inherited neuromuscular disorders. We are also pursuing basic mechanistic studies related to two such disorders: Facioscapulohumeral Muscular Dystrophy (FSHD) and Limb-Girdle Muscular Dystrophy Type 1A (LGMD1A). We use a broad range of tools, including molecular techniques, biochemistry, viral vectors, and mouse models of disease.
- J Liu, LM Wallace, SE Garwick-Coppens, D Sloboda, C Davis, CH Hakim, ME Hauser, JR Mendell, SV Brooks, and SQ Harper. RNAi-mediated gene silencing of mutant myotilin improves myopathy in LGMD1A mice. Molecular Therapy Nucleic Acids Apr 29;3e160, 2014. PMID: 24781192.
- U.S. Patents 8,481,710 and 8,524,879. Inventors: BL Davidson, H Xia, Q Mao, H Paulson, RL Boudreau, and SQ Harper. “RNA interference suppression of neurodegenerative diseases and methods of use thereof”. Issued July 9, 2013 and Sept 3, 2013.
- 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.
- 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.
- 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.
- 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.
- S.Q. Harper. Progress and challenges in RNA interference therapy for Huntington disease (2009) Archives of Neurology, 66(8): 933-938.
- L.M. Wallace, S.E. Garwick, W. Mei, A. Belayew, F. Coppee, K.J. Ladner, D. Guttridge, J. Yang, and S.Q. Harper. DUX4, a candidate gene for Facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo. (2010) Annals of Neurology, (2011) Epub Oct 28; March;69(3):540-52.
- L.M. Wallace, S.E. Garwick-Coppens, R. Rupler, and S.Q. Harper. RNA interference improves myopathic phenotypes in mice over-expressing FSHD Region Gene 1 (FRG1) (2011) Molecular Therapy, Nov;19(11):2048-54.
- L.M. Wallace, J. Liu, J.S. Domire, S.E. Garwick-Coppens, S.M. Guckes, J.R. Mendell, K.M. Flanigan, and S.Q. Harper. RNA interference inhibits DUX4-induced muscle toxicity in vivo: Implications for a targeted FSHD therapy (2012) Molecular Therapy July:20(7):1417-23.
- J. Liu and S.Q. Harper. (2012) RNAi-based gene therapy strategies for dominant limb girdle muscular dystrophies. Current Gene Therapy 12(4):307-314.
- L.M. Wallace, A. Moreo, K.R. Clark, and S.Q. Harper. Dose dependent toxicity of humanized Renilla reniformis GFP (hrGFP) limits its utility as a reporter gene in mouse muscle (2013) In press at Molecular Therapy Nucleic Acids.