Results 171 to 180 of about 106,524 (297)

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Science, 2016
Christopher E. Nelson, Chady H. Hakim, David G. Ousterout, P. Thakore, E. A. Moreb, R. Rivera, S. Madhavan, Xiufang Pan, F. Ran, Winston X. Yan, A. Asokan, Feng Zhang, D. Duan, C. Gersbach   +13 more
semanticscholar   +1 more source

Domiciliary nasal respiratory support : first experiences in Malta [PDF]

, 1997
Nasal respiratory support is a non-invasive alternative to conventional assisted ventilation with endotracheal intubation, or the more cumbersome negative pressure ventilators.
Camilleri, R., Galea Debono, Anthony, Montefort, Stephen   +2 more
core  

CSE/H2S/SESN2 Signalling Mediates the Protective Effect of Exercise Against Immobilization‐Induced Muscle Atrophy in Mice

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 5, October 2025.
ABSTRACT Background Hydrogen sulphide (H2S), a gasotransmitter synthesized by cystathionine‐γ‐lyase (CSE), exhibits antioxidant properties and may mimic exercise‐induced muscle protection. However, its mechanistic role in muscle atrophy and exercise intervention remains unclear.
Xiuru Li, Yating Huang, Xuege Yang, Sujuan Liu, Yanmei Niu, Li Fu   +5 more
wiley   +1 more source

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

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

Quantitative Muscle Ultrasound: A Non‐Invasive Biomarker for Monitoring Duchenne Muscular Dystrophy

Muscle &Nerve, Volume 72, Issue 4, Page 606-615, October 2025.
ABSTRACT Introduction/Aims Quantitative muscle ultrasound (QMUS) shows promise as a non‐invasive biomarker for monitoring functional status in Duchenne muscular dystrophy (DMD). We evaluated the correlation between QMUS in various muscles and functional capacity assessments.
Yu Jin Im, Yumi Choe, Jiwon Lee, Jeehun Lee, Jong Geol Do, Jeong‐Yi Kwon   +5 more
wiley   +1 more source

Elucidating the Role and Mechanism of Alpha‐Enolase in Senescent Amelioration via Metabolic Reprogramming

Cell Proliferation, Volume 58, Issue 10, October 2025.
KB2764 interacted with alpha‐enolase (ENO1) and pyruvate kinase M (PKM), ultimately leading to PKM phosphorylation of ENO1. KB2764 consequently increased mitochondrial ATP production and decreased glycolysis dependence. Furthermore, the action of KB2764 extends its application to extend the lifespan of Caenorhabditis elegans.
Yun Haeng Lee, Hyunwoong Lim, Gyungmin Kim, Geonhee Jang, Myeong Uk Kuk, Ji Ho Park, Jee hee Yoon, Yoo Jin Lee, Duyeol Kim, Byeonghyeon So, Minseon Kim, Hyung Wook Kwon, Youngjoo Byun, Joon Tae Park   +13 more
wiley   +1 more source

[A predictive analysis of the association between clinical phenotypes and genotypes in children with Becker muscular dystrophy/Duchenne muscular dystrophy]. [PDF]

Zhongguo Dang Dai Er Ke Za Zhi, 2020
Niu HH, Tao DY, Cheng SQ.
europepmc   +1 more source

Parental perspectives on family‐centered care in pediatric neurology: An explanatory sequential mixed‐methods study

Developmental Medicine &Child Neurology, Volume 67, Issue 10, Page 1340-1353, October 2025.
The proposed conceptual framework highlighting parents' central role in their child's healthcare experience. This original article is commented by Kettle‐Frisby on pages 1247–1248 of this issue. Abstract Aim To explore parents' experiences of family‐centered care (FCC) in a pediatric neurology clinic. Method In this explanatory sequential mixed‐methods
Ege Sarikaya, Courtney B. Cook, Kathryn A. Selby, Ye Shen, GenCOUNSEL Study, Alison M. Elliott, Jehannine Austin, Bartha Knoppers, Larry D. Lynd, Alivia Dey, Shelin Adam, Nick Bansback, Patricia Birch, Lorne Clarke, Nick Dragojlovic, Jan Friedman, Debby Lambert, Daryl Pullman, Alice Virani, Wyeth Wasserman, Ma’n Zawati, Alison M. Elliott   +21 more
wiley   +1 more source

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

Lancet Neurology, 2018
D. Birnkrant, K. Bushby, C. Bann, S. Apkon, Angela Blackwell, D. Brumbaugh, L. Case, P. Clemens, S. Hadjiyannakis, S. Pandya, N. Street, J. Tomezsko, K. Wagner, L. Ward, D. Weber   +14 more
semanticscholar   +1 more source