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Collaborations and top research areas from the last five years
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MDSRC Training Core
Harper, S. Q. (PI)
Eunice Kennedy Shriver National Institute of Child Health and Human Development
08/1/25 → 07/31/26
Project: Research
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MDSRC for Next Directions in Muscular Dystrophy Research
Flanigan, K. M. (PI), Wein, N. (CoPI), Saad, N. (CoPI), Martin, P. T. (CoPI) & Harper, S. Q. (CoPI)
Eunice Kennedy Shriver National Institute of Child Health and Human Development
08/15/24 → 07/31/26
Project: Research
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tRNA Gene Replacement Therapy for tRNA-Synthetase Related Charcot-Marie-Tooth Disorder
Burgess, R. R. W. (PI) & Harper, S. Q. (CoPI)
NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
07/1/24 → 06/30/26
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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Synaptic dysregulation in a mouse model of GRIN2D developmental and epileptic encephalopathy
Teoh, J. J., Simko, J., Camp, C. R., Liu, C. J., Wang, W., Williams, D. J., Ma, L., Soundararajan, D., Martin, C., Taylor, N. K., François, E., Petri, S., Kanber, A., Ravichandra, A., Pero, M. E., Bartolini, F., Swayne, T. C., Lutz, C. M., Zuberi, A. & Rubinstein, M. & 8 others, , Nov 1 2025, In: Brain. 148, 11, p. 3973-3988 16 p.Research output: Contribution to journal › Article › peer-review
2 Link opens in a new tab Scopus citations -
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 Access13 Link opens in a new tab 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 Access13 Link opens in a new tab 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 Access3 Link opens in a new tab 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 Access86 Link opens in a new tab Scopus citations