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Scott Q. Harper, PhD - Publications

Affiliations: 
Pediatrics and Center for Gene Therapy The Ohio State University and Nationwide Children's Hospital 
Area:
muscle gene therapy, RNAi, FSHD, LGMD1A, DMD
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66 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2024 Jones DJ, Soundararajan D, Taylor NK, Aimiuwu OV, Mathkar P, Shore A, Teoh JJ, Wang W, Sands TT, Weston MC, Harper SQ, Frankel WN. Effective knockdown - replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 39127888 DOI: 10.1016/j.ymthe.2024.08.009  0.316
2022 Stavrou M, Kagiava A, Choudury SG, Jennings MJ, Wallace LM, Fowler AM, Heslegrave A, Richter J, Tryfonos C, Christodoulou C, Zetterberg H, Horvath R, Harper SQ, Kleopa KA. A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice. The Journal of Clinical Investigation. PMID 35579942 DOI: 10.1172/JCI159814  0.32
2021 Saad NY, Al-Kharsan M, Garwick-Coppens SE, Chermahini GA, Harper MA, Palo A, Boudreau RL, Harper SQ. Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy. Nature Communications. 12: 7128. PMID 34880230 DOI: 10.1038/s41467-021-27430-1  0.662
2021 Bal NC, Gupta SC, Pant M, Sopariwala DH, Gonzalez-Escobedo G, Turner J, Gunn JS, Pierson CR, Harper SQ, Rafael-Fortney JA, Periasamy M. Is Upregulation of Sarcolipin Beneficial or Detrimental to Muscle Function? Frontiers in Physiology. 12: 633058. PMID 33732165 DOI: 10.3389/fphys.2021.633058  0.305
2021 Rashnonejad A, Amini-Chermahini G, Taylor NK, Wein N, Harper SQ. Designed U7 snRNAs inhibit  expression and improve FSHD-associated outcomes in  overexpressing cells and FSHD patient myotubes. Molecular Therapy. Nucleic Acids. 23: 476-486. PMID 33510937 DOI: 10.1016/j.omtn.2020.12.004  0.393
2020 Aimiuwu OV, Fowler AM, Sah M, Teoh JJ, Kanber A, Pyne NK, Petri S, Rosenthal-Weiss C, Yang M, Harper SQ, Frankel WN. RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 32353324 DOI: 10.1016/J.Ymthe.2020.04.007  0.425
2020 Morelli KH, Hatton CL, Harper SQ, Burgess RW. Gene Therapies for Axonal Neuropathies: Available Strategies, Successes to Date, and What to Target Next. Brain Research. 146683. PMID 32001243 DOI: 10.1016/J.Brainres.2020.146683  0.436
2019 Morelli KH, Griffin LB, Pyne NK, Wallace LM, Fowler AM, Oprescu SN, Takase R, Wei N, Meyer-Schuman R, Mellacheruvu D, Kitzman JO, Kocen SG, Hines TJ, Spaulding EL, Lupski JR, ... ... Harper SQ, et al. Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models. The Journal of Clinical Investigation. PMID 31557132 DOI: 10.1172/Jci130600  0.424
2019 Amini-Chermahini G, Rashnonejad A, Harper SQ. RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro. Rna (New York, N.Y.). PMID 31209064 DOI: 10.1261/Rna.070177.118  0.404
2019 Chermahini GA, Rashnonejad A, Harper S. P.50An in situ hybridization-based method for detecting DUX4 RNA expression in vitro Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.079  0.327
2019 Wallace L, Chermahini GA, Fowler A, Guggenbiller M, Kazimir K, Cash C, Knoblaugh S, Harper S. P.49Translating DUX4-targeted RNAi-based gene therapy for FSHD Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.078  0.376
2019 Rashnonejad A, Chermahini GA, Wallace L, Harper S. O.8DUX4 mRNA silencing with CRISPR-Cas13 gene therapy as a prospective treatment for Facioscapulohumeral muscular dystrophy Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.026  0.355
2018 Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, ... ... Harper SQ, et al. AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD. Jci Insight. 3. PMID 30429376 DOI: 10.1172/Jci.Insight.123538  0.506
2018 Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Molecular Therapy. Methods & Clinical Development. 8: 121-130. PMID 29387734 DOI: 10.1016/J.Omtm.2017.12.005  0.439
2017 Ansseau E, Vanderplanck C, Wauters A, Harper SQ, Coppée F, Belayew A. Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD). Genes. 8. PMID 28273791 DOI: 10.3390/Genes8030093  0.452
2017 Wallace L, Griffin D, Pyne N, Domire J, Rodino-Klapac L, Harper S. P.340 - Translating DUX4-targeted RNAi therapy for Facioscapulohumeral muscular dystrophy Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.380  0.325
2016 Eidahl JO, Giesige CR, Domire JS, Wallace LM, Fowler AM, Guckes SM, Garwick-Coppens SE, Labhart P, Harper SQ. Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4. Human Molecular Genetics. 25: 4577-4589. PMID 28173143 DOI: 10.1093/Hmg/Ddw287  0.464
2016 Eidahl JO, Giesige CR, Domire JS, Wallace LM, Fowler AM, Guckes S, Garwick-Coppens S, Labhart P, Harper SQ. Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4. Human Molecular Genetics. PMID 27616568 DOI: 10.1093/hmg/ddw287  0.362
2016 Ansseau E, Eidahl JO, Lancelot C, Tassin A, Matteotti C, Yip C, Liu J, Leroy B, Hubeau C, Gerbaux C, Cloet S, Wauters A, Zorbo S, Meyer P, Pirson I, ... ... Harper SQ, et al. Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation. Plos One. 11: e0146893. PMID 26816005 DOI: 10.1371/Journal.Pone.0146893  0.391
2016 Wallace LM, Giesige CR, Griffin DA, Rodino-Klapaca LR, Harper SQ. 625. RNAi Therapy for Dominant Limb Girdle Muscular Dystrophy Type 1A Molecular Therapy. 24: S248. DOI: 10.1016/S1525-0016(16)33433-5  0.52
2016 Wallace LM, Domire JS, Griffin DA, Rodino-Klapac LR, Harper SQ. 386. Toxicology for DUX4-Targeted MicroRNAs Molecular Therapy. 24: S153. DOI: 10.1016/S1525-0016(16)33195-1  0.508
2015 Ansseau E, Domire JS, Wallace LM, Eidahl JO, Guckes SM, Giesige CR, Pyne NK, Belayew A, Harper SQ. Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences. Plos One. 10: e0118813. PMID 25742305 DOI: 10.1371/Journal.Pone.0118813  0.433
2015 Wallace LM, Domire JS, Harper SQ. 610. Translating DUX4-Targeted RNAi Therapy for FSHD Molecular Therapy. 23: S242. DOI: 10.1016/S1525-0016(16)34219-8  0.503
2014 Liu J, Wallace LM, Garwick-Coppens SE, Sloboda DD, Davis CS, Hakim CH, Hauser MA, Brooks SV, Mendell JR, Harper SQ. RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice. Molecular Therapy. Nucleic Acids. 3: e160. PMID 24781192 DOI: 10.1038/Mtna.2014.13  0.551
2014 O'Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, et al. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013. Human Gene Therapy. 25: 488-97. PMID 24773122 DOI: 10.1089/Hum.2014.045  0.354
2014 Wallace L, Liu J, Garwick-Coppens S, Guckes S, Smith C, McBride J, Harper S. G.P.10 Neuromuscular Disorders. 24: 797. DOI: 10.1016/J.Nmd.2014.06.024  0.649
2013 Wallace LM, Moreo A, Clark KR, Harper SQ. Dose-dependent Toxicity of Humanized Renilla reniformis GFP (hrGFP) Limits Its Utility as a Reporter Gene in Mouse Muscle. Molecular Therapy. Nucleic Acids. 2: e86. PMID 23591809 DOI: 10.1038/Mtna.2013.16  0.526
2013 Harper SQ. Molecular dissection of dystrophin identifies the docking site for nNOS. Proceedings of the National Academy of Sciences of the United States of America. 110: 387-8. PMID 23277550 DOI: 10.1073/Pnas.1220256110  0.361
2013 Wallace L, Liu J, Garwick-Coppens S, Guckes S, Harper S. P.16.2 The DUX4 promoter is expressed in FSHD-affected tissues Neuromuscular Disorders. 23: 823. DOI: 10.1016/J.Nmd.2013.06.639  0.448
2013 Domire J, Wallace L, Guckes S, Liu J, Harper S. P.16.1 DUX4 regulates expression of the Pro-Apoptotic Gene, p63 Neuromuscular Disorders. 23: 822. DOI: 10.1016/J.Nmd.2013.06.638  0.446
2012 Pandey SN, Cabotage J, Shi R, Dixit M, Sutherland M, Liu J, Muger S, Harper SQ, Nagaraju K, Chen YW. Conditional over-expression of PITX1 causes skeletal muscle dystrophy in mice. Biology Open. 1: 629-639. PMID 23125914 DOI: 10.1242/Bio.20121305  0.426
2012 Liu J, Harper SQ. RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies. Current Gene Therapy. 12: 307-14. PMID 22856606 DOI: 10.2174/156652312802083585  0.459
2012 Wallace LM, Liu J, Domire JS, Garwick-Coppens SE, Guckes SM, Mendell JR, Flanigan KM, Harper SQ. RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1417-23. PMID 22508491 DOI: 10.1038/Mt.2012.68  0.514
2012 Liu J, Wallace L, Garwick-Coppens S, Hauser M, Mendell J, Harper S. T.O.1 RNAi therapy for LGMD1A Neuromuscular Disorders. 22: 906. DOI: 10.1016/J.Nmd.2012.06.338  0.523
2012 Wallace L, Liu J, Harper S. G.P.110 The DUX4 promoter is preferentially expressed in FSHD-affected tissues Neuromuscular Disorders. 22: 901. DOI: 10.1016/J.Nmd.2012.06.323  0.458
2012 Domire J, Coppens S, Wallace L, Guckes S, Harper S. G.P.109 Elucidation of DUX4 binding site affinities by SELEX-Seq Neuromuscular Disorders. 22: 901. DOI: 10.1016/J.Nmd.2012.06.322  0.357
2011 Garwick-Coppens SE, Herman A, Harper SQ. Construction of permanently inducible miRNA-based expression vectors using site-specific recombinases. Bmc Biotechnology. 11: 107. PMID 22087765 DOI: 10.1186/1472-6750-11-107  0.344
2011 Wallace LM, Garwick-Coppens SE, Tupler R, Harper SQ. RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1). Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 2048-54. PMID 21730972 DOI: 10.1038/Mt.2011.118  0.526
2011 Wallace LM, Garwick SE, Mei W, Belayew A, Coppee F, Ladner KJ, Guttridge D, Yang J, Harper SQ. DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo. Annals of Neurology. 69: 540-52. PMID 21446026 DOI: 10.1002/Ana.22275  0.407
2011 Boudreau RL, Garwick-Coppens SE, Liu J, Wallace LM, Harper SQ. Rapid cloning and validation of MicroRNA shuttle vectors: A practical guide Neuromethods. 58: 19-37. DOI: 10.1007/978-1-61779-114-7_2  0.593
2009 Blankinship MJ, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, Miller AD, Chamberlain JS. Erratum to "Efficient Transduction of Skeletal Muscle Using Vectors Based on Adeno-associated Virus Serotype 6". Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1482. PMID 28160909 DOI: 10.1038/Mt.2009.135  0.752
2009 Harper SQ. Progress and challenges in RNA interference therapy for Huntington disease. Archives of Neurology. 66: 933-8. PMID 19667213 DOI: 10.1001/Archneurol.2009.180  0.394
2008 Packer AN, Xing Y, Harper SQ, Jones L, Davidson BL. The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 28: 14341-6. PMID 19118166 DOI: 10.1523/Jneurosci.2390-08.2008  0.501
2008 McBride JL, Boudreau RL, Harper SQ, Staber PD, Monteys AM, Martins I, Gilmore BL, Burstein H, Peluso RW, Polisky B, Carter BJ, Davidson BL. 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. 105: 5868-73. PMID 18398004 DOI: 10.1073/Pnas.0801775105  0.764
2008 Harper SQ, Gonzalez-Alegre P. Lentivirus-mediated RNA interference in mammalian neurons. Methods in Molecular Biology (Clifton, N.J.). 442: 95-112. PMID 18369781 DOI: 10.1007/978-1-59745-191-8_8  0.389
2008 Fineberg SK, Ghosh LL, He B, Harper SQ, Davidson BL. miR-34a modulates neural progenitor cell differentiation Developmental Biology. 319: 535. DOI: 10.1016/J.Ydbio.2008.05.251  0.641
2006 Allaire PD, Ritter B, Thomas S, Burman JL, Denisov AY, Legendre-Guillemin V, Harper SQ, Davidson BL, Gehring K, McPherson PS. Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 26: 13202-12. PMID 17182770 DOI: 10.1523/Jneurosci.4608-06.2006  0.471
2006 Harper SQ, Staber PD, Beck CR, Fineberg SK, Stein C, Ochoa D, Davidson BL. Optimization of feline immunodeficiency virus vectors for RNA interference. Journal of Virology. 80: 9371-80. PMID 16973543 DOI: 10.1128/Jvi.00958-06  0.744
2006 Mas-Monteys A, Harper SQ, Gilmore BL, Staber PD, Schaffer C, Polisky B, Vargeese C, Davidson BL. 711. Allele-Specific Silencing of Mutant Huntingtin for Huntington's Disease Therapy Molecular Therapy. 13: S274-S275. DOI: 10.1016/J.Ymthe.2006.08.790  0.552
2006 McBride JL, Harper SQ, Staber PD, Martins IH, Burstein H, Peluso RW, Polisky B, Carter BJ, Davidson BL. 414. Non-Allele Specific RNA Interference in the CAG140 Knock-In Mouse Model of Huntington's Disease Molecular Therapy. 13: S159. DOI: 10.1016/J.Ymthe.2006.08.478  0.683
2006 Harper SQ, Staber PD, Rowley C, Fineberg S, Ochoa D, Stein C, Davidson BL. 371. Optimization of Feline Immunodeficiency Viral Vectors for RNA Interference Molecular Therapy. 13: S141. DOI: 10.1016/J.Ymthe.2006.08.430  0.737
2006 Staber PD, Vargeese C, Martins IH, Polisky B, Mas-Monteys A, Harper SQ, Davidson BL. 88. Synthetic siRNAs for Huntington's Disease Therapy Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.106  0.515
2005 Miller VM, Nelson RF, Gouvion CM, Williams A, Rodriguez-Lebron E, Harper SQ, Davidson BL, Rebagliati MR, Paulson HL. CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 25: 9152-61. PMID 16207874 DOI: 10.1523/Jneurosci.3001-05.2005  0.637
2005 Harper SQ, Davidson BL. Plasmid-based RNA interference: construction of small-hairpin RNA expression vectors. Methods in Molecular Biology (Clifton, N.J.). 309: 219-35. PMID 15990403 DOI: 10.1385/1-59259-935-4:219  0.523
2005 Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, Yang L, Kotin RM, Paulson HL, Davidson BL. 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. 102: 5820-5. PMID 15811941 DOI: 10.1073/Pnas.0501507102  0.6
2005 Liu M, Yue Y, Harper SQ, Grange RW, Chamberlain JS, Duan D. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Molecular Therapy : the Journal of the American Society of Gene Therapy. 11: 245-56. PMID 15668136 DOI: 10.1016/J.Ymthe.2004.09.013  0.601
2005 Davidson BL, Harper SQ. Viral delivery of recombinant short hairpin RNAs. Methods in Enzymology. 392: 145-73. PMID 15644180 DOI: 10.1016/S0076-6879(04)92009-5  0.558
2005 Harper SQ, Staber PD, He X, Martins IH, Mao Q, Paulson HL, Kotin RM, Davidson BL. 52. AAV-Delivered RNAi Improves Cellular and Motor Phenotypes in a Mouse Model for Huntington's Disease Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.080  0.611
2004 Blankinship MJ, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, Miller AD, Miller DA, Chamberlain JS. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 671-8. PMID 15451451 DOI: 10.1016/J.Ymthe.2004.07.016  0.785
2004 Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, Paulson HL, Yang L, Kotin RM, Davidson BL. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nature Medicine. 10: 816-20. PMID 15235598 DOI: 10.1038/Nm1076  0.606
2004 Xia H, Mao Q, Eliason SL, Kiewiet N, Critchfield J, Martins IH, Harper SQ, He X, Kotin RM, Zoghbi HY, Orr HT, Paulson HL, Davidson BL. 717. RNAi Therapy for Dominant Neurodegenerative Diseases Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.623  0.589
2004 Harper SQ, Staber PD, Rowley CR, He X, Martins IH, Mao Q, Paulson HL, Davidson BL. 209. Gene Silencing of Human Huntingtin Using Lentivirus-Delivered shRNA Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.150  0.617
2003 Yue Y, Li Z, Harper SQ, Davisson RL, Chamberlain JS, Duan D. Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation. 108: 1626-32. PMID 12952841 DOI: 10.1161/01.Cir.0000089371.11664.27  0.609
2002 Scott JM, Li S, Harper SQ, Welikson R, Bourque D, DelloRusso C, Hauschka SD, Chamberlain JS. Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin. Neuromuscular Disorders : Nmd. 12: S23-9. PMID 12206791 DOI: 10.1016/S0960-8966(02)00078-0  0.774
2002 Harper SQ, Crawford RW, DelloRusso C, Chamberlain JS. Spectrin-like repeats from dystrophin and alpha-actinin-2 are not functionally interchangeable. Human Molecular Genetics. 11: 1807-15. PMID 12140183 DOI: 10.1093/Hmg/11.16.1807  0.749
2002 Harper SQ, Hauser MA, DelloRusso C, Duan D, Crawford RW, Phelps SF, Harper HA, Robinson AS, Engelhardt JF, Brooks SV, Chamberlain JS. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nature Medicine. 8: 253-61. PMID 11875496 DOI: 10.1038/Nm0302-253  0.789
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