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A gene therapy approach for the treatment of limb-girdle muscular dystrophy 2C/R5.

Autor: Hermann H; University of Nevada, Reno School of Medicine, 1664 N. Virginia Avenue, Reno, NV 89557, USA., Wuebbles RD; University of Nevada, Reno School of Medicine, 1664 N. Virginia Avenue, Reno, NV 89557, USA., Burkin DJ; University of Nevada, Reno School of Medicine, 1664 N. Virginia Avenue, Reno, NV 89557, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Mar 30; Vol. 29, pp. 160-161. Date of Electronic Publication: 2023 Mar 30 (Print Publication: 2023).

Tetrahydrobiopterin shifts the balance from oxidative stress to NO signalling in Duchenne muscular dystrophy.

Autor: Wuebbles RD; Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV, USA., Oliveira-Santos A; Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV, USA., Burkin DJ; Department of Pharmacology, University of Nevada, Reno School of Medicine, Reno, NV, USA.

Publikováno v: Acta physiologica (Oxford, England) [Acta Physiol (Oxf)] 2021 Jul; Vol. 232 (3), pp. e13675. Date of Electronic Publication: 2021 May 26.

Transgenic mice expressing tunable levels of DUX4 develop characteristic facioscapulohumeral muscular dystrophy-like pathophysiology ranging in severity.

Autor: Jones TI; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Chew GL; Computational Biology Program, Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, 98109, USA.; Basic Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, 98109, USA.; Current Address: The Cancer Science Institute of Singapore, National University of Singapore, Singapore, Singapore., Barraza-Flores P; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Schreier S; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Ramirez M; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Wuebbles RD; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Burkin DJ; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA., Bradley RK; Computational Biology Program, Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, 98109, USA.; Basic Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, 98109, USA., Jones PL; Department of Pharmacology, School of Medicine, University of Nevada, Reno, Reno, NV, 89557, USA. peterjones@med.unr.edu.

Publikováno v: Skeletal muscle [Skelet Muscle] 2020 Apr 11; Vol. 10 (1), pp. 8. Date of Electronic Publication: 2020 Apr 11.

Sunitinib promotes myogenic regeneration and mitigates disease progression in the mdx mouse model of Duchenne muscular dystrophy.

Autor: Fontelonga TM; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Jordan B; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Nunes AM; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Barraza-Flores P; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Bolden N; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Wuebbles RD; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA., Griner LM; Division of Pre-clinical Innovation, NIH Center for Advancing Translational Sciences, Rockville, MD, USA., Hu X; Division of Pre-clinical Innovation, NIH Center for Advancing Translational Sciences, Rockville, MD, USA., Ferrer M; Division of Pre-clinical Innovation, NIH Center for Advancing Translational Sciences, Rockville, MD, USA., Marugan J; Division of Pre-clinical Innovation, NIH Center for Advancing Translational Sciences, Rockville, MD, USA., Southall N; Division of Pre-clinical Innovation, NIH Center for Advancing Translational Sciences, Rockville, MD, USA., Burkin DJ; Department of Pharmacology, University of Nevada, Reno School of Medicine, , Reno, NV, USA.

Publikováno v: Human molecular genetics [Hum Mol Genet] 2019 Jul 01; Vol. 28 (13), pp. 2120-2132.