Results 81 to 90 of about 7,173 (197)
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 +12 more
doaj
Frontiers in Pharmacology, 2018 Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy characterized by progressive muscle degeneration. This disease is caused by the mutation or deletion of the dystrophin gene. Currently, there are no effective treatments
Tsubasa Kameyama +20 more
doaj +1 more source
Systemic Treatment of Body‐Wide Duchenne Muscular Dystrophy Symptoms
Clinical Pharmacology &Therapeutics, Volume 116, Issue 6, Page 1472-1484, December 2024.Duchenne muscular dystrophy (DMD) is a fatal X‐linked disease that leads to premature death due to the loss of dystrophin. Current strategies predominantly focus on the therapeutic treatment of affected skeletal muscle tissue. However, certain results point to the fact that with successful treatment of skeletal muscle, DMD‐exposed latent phenotypes in ...
Patryk Konieczny
wiley +1 more source
High Throughput Screening in Duchenne Muscular Dystrophy: From Drug Discovery to Functional Genomics
Biology, 2014 Centers for the screening of biologically active compounds and genomic libraries are becoming common in the academic setting and have enabled researchers devoted to developing strategies for the treatment of diseases or interested in studying a ...
Thomas J.J. Gintjee +2 more
doaj +1 more source
The recent development, application, and future prospects of muscle atrophy animal models
MedComm – Future Medicine, Volume 3, Issue 4, December 2024.Understanding muscle atrophy: causes and models This review explores various experimental mouse models used to simulate muscle atrophy, caused by factors such as aging, chronic diseases, malnutrition, and reduced physical activity. The review emphasizes the development and limitations of these models to better understand and address the multifaceted ...
Gongchang Zhang +8 more
wiley +1 more source
Post-natal induction of PGC-1α protects against severe muscle dystrophy independently of utrophin [PDF]
, 2014 Background: Duchenne muscle dystrophy (DMD) afflicts 1 million boys in the US and has few effective treatments. Constitutive transgenic expression of the transcriptional coactivator peroxisome proliferator-activated receptor gamma coactivator (PGC)-1α ...
Arany, Zolt +5 more
core +1 more source
Regenerative biomarkers for Duchenne muscular dystrophy
Neural Regeneration Research, 2019 Skeletal muscle has an extraordinary capacity to regenerate after injury and trauma. The muscle repair mechanism is a complex process orchestrated by multiple steps.
Simon Guiraud, Kay E Davies
doaj +1 more source
Dystrophins, Utrophins, and Associated Scaffolding Complexes: Role in Mammalian Brain and Implications for Therapeutic Strategies [PDF]
, 2009 Two decades of molecular, cellular, and functional studies considerably increased our understanding of dystrophins function and unveiled the complex etiology of the cognitive deficits in Duchenne muscular dystrophy (DMD), which involves altered ...
Perronnet, Caroline, Vaillend, Cyrille
core +7 more sources
Dystrophin and utrophin: the missing links! [PDF]
, 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.
Gibson, T.J. +2 more
core +1 more source
Neurobiology of Disease, 1999 Duchenne muscular dystrophy (DMD), a severe X-linked recessive disorder which results in progressive muscle degeneration, is due to a lack of dystrophin, a membrane cytoskeletal protein.
Emmanuel Chaubourt +5 more
doaj