Personal profile
Expertise related to UN Sustainable Development Goals
In 2015, UN member states agreed to 17 global Sustainable Development Goals (SDGs) to end poverty, protect the planet and ensure prosperity for all. This person’s work contributes towards the following SDG(s):
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SDG 3 Good Health and Well-being -
SDG 9 Industry, Innovation, and Infrastructure
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Collaborations and top research areas from the last five years
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Understanding SCA1 pathogenesis in human cerebellar organoids
Cvetanovic, M. (PI), Pacak, C. A. (CoI), Vasan, G. (CoI), Dutton, J. R. (Co-Principal Investigator), Walker, K. E. (Graduate Student), McCall, A. (Other Role), Renaud, A. C. (Other Role) & Zhang, Y. (Other Role)
NIH NAT INST OF NEURO DISORDERS & STROKE
7/1/25 → 6/30/27
Project: Research project
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Canine iPSC-Cardiomyocyte Studies (TTN-DCM Program Phase II)
Pacak, C. A. (PI)
UNIVERSITY OF FLORIDA, SAREPTA THERAPEUTICS, INC.
2/21/23 → 6/8/25
Project: Research project
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Optimized Production and Validation of Rationally Designed AAV Vectors for Cockayne Syndrome Gene Therapy
Aslanidi, G. (PI), Kang, P. B. (CoI) & Pacak, C. A. (CoI)
DHHS FOOD & DRUG ADMINISTRATION (FDA)
9/1/21 → 8/31/25
Project: Research project
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Mechanisms and Treatment of Cardiac and Skeletal Muscular Dysfunction in Barth Syndrome
Pacak, C. A. (PI)
NATIONAL INSTITUTES OF HEALTH (NIH)
5/5/21 → 4/30/24
Project: Research project
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Development of HITI CRISPR gene therapy for dilated cardiomyopathy in a canine model
Pacak, C. A. (PI)
UNIVERSITY OF FLORIDA, SAREPTA THERAPEUTICS, INC.
2/1/21 → 8/31/22
Project: Research project
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The D2.B10-Dmdmdx/J Mouse Model of Duchenne Muscular Dystrophy Exhibits a Severe Mitochondrial Deficiency Not Observed in the C57BL/10ScSn-Dmdmdx/J Mouse
Tinklenberg, J. A., Sutton, J., Slick, R. A., Meng, H., Haberman, M., Prom, M. J., Beatka, M. J., Vetter, T. A., Daugherty, A. L., Pacak, C. A., Gonzalez, J. P. & Lawlor, M. W., Feb 2026, In: American Journal of Pathology. 196, 2, p. 532-549 18 p.Research output: Contribution to journal › Article › peer-review
Open Access1 Link opens in a new tab Scopus citations -
The impact of Hnrnpl deficiency on transcriptional patterns of developing muscle cells
Littel, H. R., Gunasekaran, M., Daugherty, A. L., Wells, N. M., Turner, J., Bruels, C. C., Pacak, C. A., Draper, I. & Kang, P. B., Jan 2026, In: FEBS Open Bio. 16, 1, p. 178-198 21 p.Research output: Contribution to journal › Article › peer-review
Open Access -
ACSS2 involved in acetyl-CoA synthesis regulates skeletal muscle function
Gunasekaran, M., Campos, G., Wells, N. M., Lambuu, K., Draper, I., Pacak, C. A. & Kang, P. B., Oct 2025, In: FEBS Letters. 599, 19, p. 2817-2827 11 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Cardiac Transplantation Does Not Improve Exercise Tolerance, Muscle Mass, or Substrate Metabolism in Barth Syndrome
Cade, W. T., Bohnert, K. L., Peterson, L. R., Fuentes, L. D. L., Poehlein, E., Green, C. L., Pacak, C. A., Byrne, B. J., Reeds, D. N., Bashir, A., Feingold, B. & Taylor, C., Jul 2025, In: JIMD Reports. 66, 4, e70034.Research output: Contribution to journal › Article › peer-review
Open Access1 Link opens in a new tab Scopus citations -
Effects of HMG CoA reductase (HMGCR) deficiency on skeletal muscle development
Gunasekaran, M., Littel, H. R., Wells, N. M., Turner, J., Campos, G., Venigalla, S., Estrella, E. A., Ghosh, P. S., Daugherty, A. L., Stafki, S. A., Kunkel, L. M., Foley, A. R., Donkervoort, S., Bönnemann, C. G., Toledo-Bravo de Laguna, L., Nascimento, A., Natera-de Benito, D., Draper, I., Bruels, C. C. & Pacak, C. A. & 1 others, , Sep 2025, In: FEBS Journal. 292, 18, p. 4854-4869 16 p.Research output: Contribution to journal › Article › peer-review
Open Access3 Link opens in a new tab Scopus citations
Datasets
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AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice
Chauhan, M., Daugherty, A. L., Khadir, F., Duzenli, O. F., Hoffman, A., Tinklenberg, J. A., Kang, P. B., Aslanidi, G. & Pacak, C. A., figshare, 2024
DOI: 10.6084/m9.figshare.c.7436190, https://springernature.figshare.com/collections/AAV-DJ_is_superior_to_AAV9_for_targeting_brain_and_spinal_cord_and_de-targeting_liver_across_multiple_delivery_routes_in_mice/7436190 and one more link, https://springernature.figshare.com/collections/AAV-DJ_is_superior_to_AAV9_for_targeting_brain_and_spinal_cord_and_de-targeting_liver_across_multiple_delivery_routes_in_mice/7436190/1 (show fewer)
Dataset