Scott Q. Harper, PhD
Affiliations: | Pediatrics and Center for Gene Therapy | The Ohio State University and Nationwide Children's Hospital |
Area:
muscle gene therapy, RNAi, FSHD, LGMD1A, DMDGoogle:
"Scott Harper"Parents
Sign in to add mentor| 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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Publications
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| Rashnonejad A, Farea M, Amini-Chermahini G, et al. (2024) Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b. Biorxiv : the Preprint Server For Biology |
| Jones DJ, Soundararajan D, Taylor NK, et al. (2024) 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 |
| 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.) |