Results 181 to 190 of about 59,671 (341)

Multiplex Ligation Dependent Probe Amplification Based Mutation Analysis of Dystrophin Gene in Nepalese Patients with Duchenne Muscular Dystrophy

Nepal Journal of Biotechnology, 2016
Duchenne muscular dystrophy (DMD) is X-linked recessive neuromuscular disorders caused due to mutation in dystrophin gene, leading to progressive muscle weakness.
Kushal Shrestha, Smita Shrestha, Saroj Khatiwada, Bishu Acharya, Sulochana Manandhar, Rohit Kumar Pokharel   +5 more
doaj   +1 more source

ARC‐18 Improved Motor Performance Through Inhibiting ACLY‐Mediated Smad2/3 Acetylation in a Model of Duchenne Muscular Dystrophy

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 5, October 2025.
ABSTRACT Background Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness, with inflammation and fibrosis contributing to its pathogenesis. Despite advancements in genetic disease‐modifying treatment, there is currently no effective pharmacological treatment for DMD.
Chongyang Chen, Bingge Zhang, Chao Yang, Jing Wang, Ye He, Haitao Yu, Jianjun Liu, Yongmei Xie, Xifei Yang, Gong‐Ping Liu   +9 more
wiley   +1 more source

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

Scientific Reports
Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown to stabilize or slow disease progression ...
Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac   +14 more
doaj   +1 more source

In vitro degradation of the Duchenne muscular dystrophy gene product (dystrophin)

, 1989
Kiichiro Matsumura, Teruo Shimizu, Toru Mannen   +2 more
openalex   +2 more sources

Reduced Muscle Force in Dystrophic DMDΔ52 Pigs Is Incompletely Restored by Systemic Transcript Reframing (DMDΔ51–52)

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 5, October 2025.
ABSTRACT Background Duchenne muscular dystrophy (DMD) is a fatal X‐linked disease caused by mutations in the DMD gene, leading to dystrophin deficiency and progressive degeneration of skeletal and cardiac muscles. Pigs lacking DMD exon 52 (DMDΔ52) are a clinically severe model for DMD, mimicking molecular, functional and pathological hallmarks of the ...
Michaela Blasi, Hristiyan Hristov, Jan B. Stöckl, Martin Kraetzl, Sonja Fiedler, Elisabeth Kemter, Mayuko Kurome, Barbara Kessler, Josep M. Cambra, Valeri Zakhartchenko, Maggie C. Walter, Christian Kupatt, Nikolai Klymiuk, Thomas Fröhlich, Andreas Blutke, Michael Stirm, Florian Jaudas, Eckhard Wolf   +17 more
wiley   +1 more source

Dystrophin expression in the mdx mouse restored by stem cell transplantation

Nature, 1999
E. Gussoni, Y. Soneoka, Corinne D. Strickland, E. Buzney, Mohamed K. Khan, A. Flint, L. Kunkel, R. Mulligan   +7 more
semanticscholar   +1 more source

Gene and RNA Editing: Revolutionary Approaches to Treating Diseases

MedComm, Volume 6, Issue 10, October 2025.
The image illustrates gene editing technologies: DNA editing using CRISPR–Cas9 and RNA editing via Cas13d, with their clinical applications and ethical risks. DNA editing allows precise gene modifications for conditions like amyotrophic lateral sclerosis [ALS] and Huntington's disease, while RNA editing supports multiplexed modifications.
Jia‐Mei Li, Jie Huang, Yan Liao, Ting Hu, Chang‐Li Wang, Wang‐Zheqi Zhang, Chen‐Wei Huang   +6 more
wiley   +1 more source

Temporal Bayesian classifiers for modelling muscular dystrophy expression data [PDF]

, 2006
The analysis of microarray data from time-series experiments requires specialised algorithms, which take the temporal ordering of the data into account. In this paper we explore a new architecture of Bayesian classifier that can be used to understand how
Hoen, PAC't, Liu, X, Tucker, A, Vinciotti, V   +3 more
core  

Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy.

Physiological Reviews, 2016
D. Allen, N. Whitehead, S. Froehner
semanticscholar   +1 more source

Heart Regeneration and Repair: Molecular Mechanism and Therapeutic Targets

MedComm, Volume 6, Issue 10, October 2025.
Regarding the general picture of heart regeneration therapy, it encompasses exogenous cell therapy, endogenous medications, metabolism, interventions in gene pathways, as well as the therapeutic strategies in the burgeoning interdisciplinary field of medicine and engineering in recent years.
Mingchuan Liu, Tingwei Peng, Rui Yu, Kexin Wang, Di Wang, Xiaojie Jia, Yan Zhang, Jianqiang Hu, Bingchao Qi, Yan Li   +9 more
wiley   +1 more source