Projects per year
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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Collaborations and top research areas from the last five years
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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 → 2/20/26
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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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, , 2025, (Accepted/In press) In: FEBS Journal.Research output: Contribution to journal › Article › peer-review
Open Access -
Rescue of mitochondrial dysfunction through alteration of extracellular matrix composition in barth syndrome cardiac fibroblasts
Piñeiro-Llanes, J., Suzuki-Hatano, S., Jain, A., Venigalla, S., Kamat, M., Basso, K. B., Cade, W. T., Simmons, C. S. & Pacak, C. A., Apr 2025, In: Biomaterials. 315, 122922.Research output: Contribution to journal › Article › peer-review
Open Access -
The mouse Social Frailty Index (mSFI): a novel behavioral assessment for impaired social functioning in aging mice
Collinge, C. W., Razzoli, M., Mansk, R., McGonigle, S., Lamming, D. W., Pacak, C. A., van der Pluijm, I., Niedernhofer, L. & Bartolomucci, A., Feb 2025, In: GeroScience. 47, 1, p. 85-107 23 p., e2607.Research output: Contribution to journal › Article › peer-review
Open Access -
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., Dec 2024, In: Journal of Translational Medicine. 22, 1, 824.Research output: Contribution to journal › Article › peer-review
Open Access -
Cognitive Decline and Other Late-Stage Neurologic Complications in Cockayne Syndrome
Rajamani, G., Stafki, S. A., Daugherty, A. L., Mantyh, W. G., Littel, H. R., Bruels, C. C., Pacak, C. A., Robbins, P. D., Niedernhofer, L. J., Abiona, A., Giunti, P., Mohammed, S., Laugel, V. & Kang, P. B., May 16 2024, In: Neurology: Clinical Practice. 14, 4, e200309.Research output: Contribution to journal › Article › peer-review
1 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
Dataset
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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.v1, 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
Dataset