Results 41 to 50 of about 7,758 (250)

PMO-based let-7c site blocking oligonucleotide (SBO) mediated utrophin upregulation in mdx mice, a therapeutic approach for Duchenne muscular dystrophy (DMD)

Scientific Reports, 2020
Upregulation of utrophin, a dystrophin related protein, is considered a promising therapeutic approach for Duchenne muscular dystrophy (DMD). Utrophin expression is repressed at the post-transcriptional level by a set of miRNAs, among which let-7c is ...
Kasturi Sengupta, Emanuele Loro, Tejvir S. Khurana   +2 more
doaj   +1 more source

Inactivation of Sirt6 ameliorates muscular dystrophy in mdx mice by releasing suppression of utrophin expression

Nature Communications, 2022
Utrophin is a dystrophin-related protein stabilizing the sarcolemma in absence of dystrophin. Here the authors report that inactivation of the protein deacetylase SIRT6, involved in the deacetylation of the epigenetic mark H3K56ac in muscle cells ...
Angelina M. Georgieva, Xinyue Guo, Marek Bartkuhn, Stefan Günther, Carsten Künne, Christian Smolka, Ann Atzberger, Ulrich Gärtner, Kamel Mamchaoui, Eva Bober, Yonggang Zhou, Xuejun Yuan, Thomas Braun   +12 more
doaj   +1 more source

Sarcospan Regulates Cardiac Isoproterenol Response and Prevents Duchenne Muscular Dystrophy-Associated Cardiomyopathy. [PDF]

, 2015
BackgroundDuchenne muscular dystrophy is a fatal cardiac and skeletal muscle disease resulting from mutations in the dystrophin gene. We have previously demonstrated that a dystrophin-associated protein, sarcospan (SSPN), ameliorated Duchenne muscular ...
Crosbie-Watson, Rachelle H, Jordan, Maria C, Marshall, Jamie L, Nguyen, Reginald T, Parvatiyar, Michelle S, Richardson, Vanitra A, Roos, Kenneth P   +6 more
core   +1 more source

Duchenne muscular dystrophy cell culture models created by CRISPR/Cas9 gene editing and their application in drug screening

Scientific Reports, 2021
Gene editing methods are an attractive therapeutic option for Duchenne muscular dystrophy, and they have an immediate application in the generation of research models. To generate myoblast cultures that could be useful in in vitro drug screening, we have
Patricia Soblechero-Martín, Edurne Albiasu-Arteta, Aina Anton-Martinez, Laura de la Puente-Ovejero, Iker Garcia-Jimenez, Gabriela González-Iglesias, Irene Larrañaga-Aiestaran, Andrea López-Martínez, Javier Poyatos-García, Estíbaliz Ruiz-Del-Yerro, Federico Gonzalez, Virginia Arechavala-Gomeza   +11 more
doaj   +1 more source

Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model

Molecular Therapy: Methods & Clinical Development, 2023
Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are currently in clinical trials, but have not been thoroughly investigated for their efficacy on cardiomyopathy progression to heart failure. We previously validated Fiona/
Arden B. Piepho, Jeovanna Lowe, Laurel R. Cumby, Lisa E. Dorn, Dana M. Lake, Neha Rastogi, Megan D. Gertzen, Sarah L. Sturgill, Guy L. Odom, Mark T. Ziolo, Federica Accornero, Jeffrey S. Chamberlain, Jill A. Rafael-Fortney   +12 more
doaj   +1 more source

Dystrophin and utrophin: the missing links!

FEBS Letters, 1995
There is considerable sequence homology between dystrophin and utrophin, both at the protein and DNA level, and consequently it was assumed that their domain structures and functions would be similar. As more of the detailed biochemical and cell biological properties of these two proteins become known, so it becomes clear that there are subtle if not ...
Steven J. Winder, Toby J. Gibson, John Kendrick-Jones   +2 more
openaire   +3 more sources

Molecular and Functional Analysis of the Utrophin Promoter [PDF]

Nucleic Acids Research, 1996
Utrophin is a ubiquitously expressed cytoskeletal protein which is an important structural component of the mammalian neuromuscular junction. It shows extensive sequence similarity to dystrophin leading to postulation that utrophin may be able to compensate for the absence of dystrophin in Duchenne muscular dystrophy (DMD) patients.
Jonathon M. Tinsley, Kay E. Davies, Anne E. Deconinck, Carina L. Dennis   +3 more
openaire   +3 more sources

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene [PDF]

Gene Therapy, 2000
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a subsarcolemmal protein, dystrophin. We have previously shown that the dystrophin-related protein, utrophin is able to compensate for the lack of dystrophin in the mdx mouse, the mouse model for DMD.
Wakefield, P. M., Tinsley, J. M., Wood, M. J. A., Gilbert, R., Karpati, G., Davies, K. E.   +5 more
openaire   +5 more sources

Modulation of PGC-1α activity as a treatment for metabolic and muscle-related diseases [PDF]

, 2014
Physical inactivity is a predisposing factor for various disease states including obesity, cardiovascular disease, as well as for certain types of cancer. Regular endurance exercise mediates several beneficial effects such as increased energy expenditure
Handschin, Christoph, Svensson, Kristoffer   +1 more
core   +1 more source

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice. [PDF]

PLoS ONE, 2010
Inactivation of all utrophin isoforms in dystrophin-deficient mdx mice results in a strain of utrophin knockout mdx (uko/mdx) mice. Uko/mdx mice display severe clinical symptoms and die prematurely as in Duchenne muscular dystrophy (DMD) patients.
Dejia Li, Yongping Yue, Dongsheng Duan
doaj   +1 more source