Scott Q. Harper, PhD

Pediatrics and Center for Gene Therapy The Ohio State University and Nationwide Children's Hospital 
muscle gene therapy, RNAi, FSHD, LGMD1A, DMD
"Scott Harper"


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Jeffrey Chamberlain grad student 1997-2002 University of Michigan
 (Structural flexibilty of the dystrophin rod domain: Implications for gene therapy of Duchenne muscular dystrophy using adeno -associated viral vectors.)
Beverly L. Davidson post-doc 2002-2007
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Stavrou M, Kagiava A, Choudury SG, et al. (2022) A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice. The Journal of Clinical Investigation
Saad NY, Al-Kharsan M, Garwick-Coppens SE, et al. (2021) Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy. Nature Communications. 12: 7128
Bal NC, Gupta SC, Pant M, et al. (2021) Is Upregulation of Sarcolipin Beneficial or Detrimental to Muscle Function? Frontiers in Physiology. 12: 633058
Rashnonejad A, Amini-Chermahini G, Taylor NK, et al. (2021) Designed U7 snRNAs inhibit  expression and improve FSHD-associated outcomes in  overexpressing cells and FSHD patient myotubes. Molecular Therapy. Nucleic Acids. 23: 476-486
Aimiuwu OV, Fowler AM, Sah M, et al. (2020) 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
Morelli KH, Hatton CL, Harper SQ, et al. (2020) Gene Therapies for Axonal Neuropathies: Available Strategies, Successes to Date, and What to Target Next. Brain Research. 146683
Morelli KH, Griffin LB, Pyne NK, et al. (2019) Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models. The Journal of Clinical Investigation
Amini-Chermahini G, Rashnonejad A, Harper SQ. (2019) RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro. Rna (New York, N.Y.)
Chermahini GA, Rashnonejad A, Harper S. (2019) P.50An in situ hybridization-based method for detecting DUX4 RNA expression in vitro Neuromuscular Disorders. 29
Wallace L, Chermahini GA, Fowler A, et al. (2019) P.49Translating DUX4-targeted RNAi-based gene therapy for FSHD Neuromuscular Disorders. 29
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