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Dive into the research topics where Scott Q. Harper is active. These topic labels come from the works of this person. Together they form a unique fingerprint.
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Collaborations and top research areas from the last five years
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tRNA Gene Replacement Therapy for tRNA-Synthetase Related Charcot-Marie-Tooth Disorder
Burgess, R. W. (PI) & Harper, S. Q. (CoPI)
National Institute of Neurological Disorders and Stroke
07/1/24 → 06/30/25
Project: Research
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AAV mediated gene knockdown of PIK3C2B as a therapeutic strategy for X-linked myotubular myopathy and fatty liver disease
Harper, S. Q. (PI) & Dowling, J. J. J. (CoPI)
National Institute of Arthritis and Musculoskeletal and Skin Diseases
07/18/23 → 06/30/25
Project: Research
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Chromatin remodeling gene therapy for FSHD using split-vector AAV SMCHD1 vectors
Harper, S. Q. (PI)
National Institute of Arthritis and Musculoskeletal and Skin Diseases
07/15/21 → 06/30/23
Project: Research
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In vivo efficacy studies to support the development of DUX4-targeted RNAi therapy for FSHD
Harper, S. Q. (PI)
National Institute of Neurological Disorders and Stroke
08/1/19 → 07/31/21
Project: Research
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A self-complementary AAV proviral plasmid that reduces cross-packaging and ITR promoter activity in AAV vector preparations
Taylor, N. K., Guggenbiller, M. J., Mistry, P. P., King, O. D. & Harper, S. Q., Sep 12 2024, In: Molecular Therapy - Methods and Clinical Development. 32, 3, 101295.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy
Jones, D. J., Soundararajan, D., Taylor, N. K., Aimiuwu, O. V., Mathkar, P., Shore, A., Teoh, J. J., Wang, W., Sands, T. T., Weston, M. C., Harper, S. Q. & Frankel, W. N., Oct 2 2024, In: Molecular Therapy. 32, 10, p. 3318-3330 13 p.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Post-Translational Modifications of the DUX4 Protein Impact Toxic Function in FSHD Cell Models
Knox, R. N., Eidahl, J. O., Wallace, L. M., Choudury, S. G., Rashnonejad, A., Daman, K., Guggenbiller, M. J., Saad, N. Y., Hoover, M. E., Zhang, L., Branson, O. E., Emerson, C. P., Freitas, M. A. & Harper, S. Q., Aug 2023, In: Annals of Neurology. 94, 2, p. 398-413 16 p.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
Coulis, G., Jaime, D., Guerrero-Juarez, C., Kastenschmidt, J. M., Farahat, P. K., Nguyen, Q., Pervolarakis, N., McLinden, K., Thurlow, L., Movahedi, S., Hughes, B. S., Duarte, J., Sorn, A., Montoya, E., Mozaffar, I., Dragan, M., Othy, S., Joshi, T., Hans, C. P. & Kimonis, V. & 15 others, , Jul 2023, In: Science Advances. 9, 27, eadd9984.Research output: Contribution to journal › Article › peer-review
Open Access33 Scopus citations -
A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice
Stavrou, M., Kagiava, A., Choudury, S. G., Jennings, M. J., Wallace, L. M., Fowler, A. M., Heslegrave, A., Richter, J., Tryfonos, C., Christodoulou, C., Zetterberg, H., Horvath, R., Harper, S. Q. & Kleopa, K. A., Jul 1 2022, In: Journal of Clinical Investigation. 132, 13, e159814.Research output: Contribution to journal › Article › peer-review
Open Access33 Scopus citations