Results 81 to 90 of about 4,882 (214)

Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling

Journal of Cachexia, Sarcopenia and Muscle, Volume 17, Issue 1, February 2026.
ABSTRACT Duchenne muscular dystrophy (DMD), caused by dystrophin deficiency, presents a multifaceted challenge that affects both skeletal muscle function and cardiomyocyte homeostasis, causing progressive degeneration and life‐threatening cardiac complications by adolescence.
Marta Przymuszała, Marta Białobrzeska, Józef Dulak, Urszula Florczyk‐Soluch   +3 more
wiley   +1 more source

Pharmacological treatments for Friedreich ataxia [PDF]

, 2016
BACKGROUND: Friedreich ataxia is a rare inherited autosomal recessive neurological disorder, characterised initially by unsteadiness in standing and walking, slowly progressing to wheelchair dependency usually in the late teens or early twenties.
Brassington, R, Fahey, M, Kearney, M, Orrell, RW, Pandolfo, M   +4 more
core  

Epigenetics and triplet-repeat neurological diseases [PDF]

, 2015
The term ‘junk DNA’ has been reconsidered following the delineation of the functional significance of repetitive DNA regions. Typically associated with centromeres and telomeres, DNA repeats are found in nearly all organisms throughout their genomes ...
Festenstein, RJ, Nageshwaran, S
core   +2 more sources

Metformin Restores Mitochondrial Function and Neurogenesis in POLG Patient‐Derived Brain Organoids

Advanced Science, Volume 13, Issue 4, 19 January 2026.
Patient‐derived POLG‐mutant cortical organoids reveal neuronal subtype‐specific mitochondrial and synaptic defects, with dopaminergic neurons most affected. Metformin treatment restores neuronal identity, mitochondrial function, and excitability, increased mtDNA maintenance, and reprogrammed metabolism via TCA and redox pathways.
Zhuoyuan Zhang, Tsering Yangzom, Ning Lu, Shenglong Deng, Xianglu Xiao, Guang Yang, Kristina Xiao Liang   +6 more
wiley   +1 more source

Fe‐S Protein FDX1 Triggers Tumor‐Intrinsic Innate Immunity via Mitochondrial Nucleic Acids Release to Orchestrate Ferroptosis in CCRCC

Advanced Science, Volume 13, Issue 6, 30 January 2026.
Restoration of the mitochondrial Fe–S protein FDX1 suppresses clear cell renal cell carcinoma by coupling ferroptosis with tumor‐intrinsic innate immune activation. FDX1 induces mitochondrial dysfunction and mtDNA/mt‐dsRNA release, activating cGAS–STING and RIG‐I/MDA5–MAVS signaling to elicit type I interferon responses, thereby promoting antigen ...
Xing Huang, Shaoqing Yu, Wenjie Wei, Wen Tao, Tianwei Cai, Lequan Wen, Jiali Ye, Chi Zhang, Huayi Feng, Senming Cao, Baojun Wang, Xin Ma, Yan Huang, Xu Zhang   +13 more
wiley   +1 more source

Coenzyme Q10 and Neurological Diseases

Pharmaceuticals, 2009
Coenzyme Q10 (CoQ10, or ubiquinone) is a small electron carrier of the mitochondrial respiratory chain with antioxidant properties. CoQ10 supplementation has been widely used for mitochondrial disorders.
Gabriele Siciliano, Anna Choub, Michelangelo Mancuso, Daniele Orsucci, Valeria Calsolaro   +4 more
doaj   +1 more source

Coenzyme Q10 Levels Are Decreased in the Cerebellum of Multiple-System Atrophy Patients [PDF]

, 2016
BACKGROUND: The objective of this study was to evaluate whether the levels of coenzyme Q10 (CoQ10) in brain tissue of multiple system atrophy (MSA) patients differ from those in elderly controls and in patients with other neurodegenerative diseases ...
Bettencourt, C, Chalasani, A, Hargreaves, I, Holton, JL, Houlden, H, Kiely, AP, Neergheen, V, Schottlaender, LV   +7 more
core   +1 more source

Polydopamine‐Based Antioxidant Countermeasures Against Spaceflight‐Induced Neurodegeneration

Small Science, Volume 6, Issue 1, January 2026.
Polydopamine nanoparticles (PDNPs) protect neuron‐like cells from microgravity‐ and cosmic radiation‐induced oxidative stress. Through in‐flight and ground‐based experiments, it has been shown that PDNPs preserve neuronal and mitochondrial function, markedly mitigating transcriptional dysregulation caused by spaceflight.
Alessio Carmignani, Attilio Marino, Matteo Battaglini, Melike Belenli Gümüş, Elisa Carrubba, Michele Balsamo, Giovanni Valentini, Gabriele Mascetti, Marco Vukich, Giada Graziana Genchi, Gianni Ciofani   +10 more
wiley   +1 more source

The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials. [PDF]

, 2015
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this disorder to properly design clinical trials.
Main, M, Manzur, AY, Mayhew, A, Mercuri, E, Muntoni, F, on behalf of UK NorthStar Clinical Network, ., Pane, M, Ricotti, V, Ridout, DA   +8 more
core   +1 more source

Nanomedicines Against Mitochondrial Dysfunction‐Induced Metabolic Diseases

Advanced Science, Volume 12, Issue 48, December 29, 2025.
Mitochondrial dysfunction is central to metabolic diseases. Nanomedicine offers transformative approaches for restoring function via targeted delivery, responsive release, and multimodal therapy. This review outlines the pathological basis, nanocarrier design, organelle‐specific targeting, recent advances, and future clinical translation challenges ...
Ke Xu, Leyi Wang, Tao Lv, Chen Jin, Qiong Wu, Yongjie Zhou, Gang Xu, Zhenyu Duan, Kui Luo, Jiayin Yang   +9 more
wiley   +1 more source