Results 91 to 100 of about 27,913 (221)

Genetic Code Expanded T Cell for Controllable Immunotherapy

Advanced Science, Volume 13, Issue 41, 22 July 2026.
Our GCE‐CAR‐T cells enables tight, dose‐dependent, and function‐preserving control of CAR expression at the translational level through amber codon suppression and genetic incorporation of ncAA. ABSTRACT Chimeric antigen receptor (CAR)‐T cell therapy has demonstrated curative potential against hematologic malignancies, but its clinical application ...
Xue Wang, Yingao Gao, Yeyu Su, Yong Wang, Tao Liu   +4 more
wiley   +1 more source

Safety considerations of gene‐based therapies for Alzheimer's disease

Alzheimer's &Dementia, Volume 22, Issue 7, July 2026.
Abstract Gene‐based therapies show increasing promise for the treatment of neurologic disease. In 2016, nusinersen, an RNA‐based therapy, was approved for children with spinal muscular atrophy (SMA). Over 200 clinical trials have utilized gene therapy approaches for a host of neurodegenerative and neuromuscular disorders, including Alzheimer's disease (
Elizabeth A. Bevins, Gabriel C. Léger, Michelle A. Herman, John M. Ringman, Subhojit Roy, Matthew Shtrahman, Judy Pa, Howard H. Feldman   +7 more
wiley   +1 more source

Health Benefits and Molecular Mechanisms of Urolithin A: From a Gut Microbiota‐Derived Metabolite to Translational Applications

Food Science &Nutrition, Volume 14, Issue 7, July 2026.
Urolithin A (UA) is a gut microbiota‐derived metabolite produced from dietary ellagitannins and ellagic acid, with endogenous formation varying among urolithin metabotypes (UM‐A, UM‐B, and UM‐0). UA regulates an interconnected network centered on mitophagy and mitochondrial quality control, antioxidant defense, inflammatory signaling, autophagy, and ...
Zijiang Yang, Chenggen Guo, Ziao Deng, Xinxuan Xue   +3 more
wiley   +1 more source

Human Disposition, Metabolism, and Excretion of Sevasemten (EDG‐5506), a Selective Modulator of Fast Myosin in Healthy Volunteers

The Journal of Clinical Pharmacology, Volume 66, Issue 7, July 2026.
Abstract Sevasemten (EDG‐5506) is a novel, orally bioavailable, investigational small molecule designed to selectively modulate type II fast skeletal myosin with the goal of protecting dystrophic muscle from contraction‐induced injury. Sevasemten is in clinical development for the treatment of Becker and Duchenne muscular dystrophy.
Molly Madden, Marc Evanchik, Jonathan Lane, Joanne Donovan, Alan J. Russell, Jeffrey A. Silverman   +5 more
wiley   +1 more source

Quantitative Muscle MRI of the Lower Extremities Reveals Different Patterns of Involvement in Classic Infantile and Young Late‐Onset Pompe Patients

Journal of Inherited Metabolic Disease, Volume 49, Issue 4, July 2026.
ABSTRACT With increased survival due to enzyme replacement therapy, children with classic infantile Pompe disease tend to develop a clinical phenotype with pronounced distal muscle weakness, while late‐onset patients typically exhibit proximal muscle weakness.
Jan J. A. van den Dorpel, Mechteld E. Guldemond, Kevin R. Keene, Pieter A. van Doorn, Ans T. van der Ploeg, Johanna M. P. van den Hout, Hermien E. Kan, Nadine A. M. E. van der Beek   +7 more
wiley   +1 more source

Cognitive Function in Duchenne Muscular Dystrophy Patients

Neurologijos seminarai
Duchenne muscular dystrophy is a rare, progressive, X-linked recessive disorder, characterized by impaired synthesis of the protein dystrophin. Motor symptoms in boys typically emerge within the first year of life, followed by progressive cardiac ...
Viktorija Urbanovič, Jurgita Grikinienė   +1 more
doaj   +1 more source

Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives

MedComm, Volume 7, Issue 7, July 2026.
Schematic illustration of four emerging CRISPR–Cas delivery platforms defined by distinct design principles and structural features: virus‐mimicking nanosystems (e.g., VLPs), cell‐derived extracellular vesicles, cell‐penetrating peptides, and stimuli‐responsive scaffolds. These platforms enable spatiotemporally controlled delivery of RNPs, mRNA, or DNA
Meijia Yang, Yiqiong Song, Ziyang Wang, Ke Chao, Lifeng Li, Xu Zhang, Xiaoran Duan, Chenglong Yu, Ruyue Xue, Jie Zhao   +9 more
wiley   +1 more source

Novel DMD Frameshift Variant (p.Leu2017Profs*5) in Spectrin‐Like Repeat 16 Expands the Mutational Spectrum of DMD

Molecular Genetics &Genomic Medicine, Volume 14, Issue 7, July 2026.
A novel DMD frameshift variant (c.6050_6051del; p.Leu2017Profs*5) causes a 2‐bp deletion in exon 42, leading to frameshift, premature truncation, and disruption of spectrin‐like repeat 16 architecture. ABSTRACT Background Duchenne muscular dystrophy (DMD) is an X‐linked neuromuscular disorder caused by pathogenic variants in the DMD gene, which encodes
Yu‐Chin Lin, G. W. Gant Luxton, Hwei‐Jen Lee, Yu‐Yang Lu, Hung‐Chi Yang, Kuo‐Sheng Hung, Ming‐Tsong Lai, Chih‐Fen Hu   +7 more
wiley   +1 more source

Asynchrony Related to Mechanical Ventilation Exacerbates Diaphragm Dysfunction in a Mouse Model of Duchenne Muscular Dystrophy

Muscle &Nerve, Volume 74, Issue 1, Page 255-264, July 2026.
ABSTRACT Introduction/Aims In dystrophic mice (mdx, a genetic homolog of Duchenne muscular dystrophy: DMD), previous studies showed that mechanical ventilation (MV) induces ventilator‐induced diaphragmatic dysfunction (VIDD). However, susceptibility to mechanical stress caused by asynchrony remains unknown.
Mohamad Yehya, Haikel Dridi, Eric Estève, Johan Moreau, Steven Reiken, Andrew R. Marks, Alain Lacampagne, Stefan Matecki   +7 more
wiley   +1 more source

Atrogin-1 promotes muscle homeostasis by regulating levels of endoplasmic reticulum chaperone BiP

JCI Insight
Skeletal muscle wasting results from numerous pathological conditions affecting both the musculoskeletal and nervous systems. A unifying feature of these pathologies is the upregulation of members of the E3 ubiquitin ligase family, resulting in increased
Avnika A. Ruparelia, Margo Montandon, Jo Merriner, Cheng Huang, Siew Fen Lisa Wong, Carmen Sonntag, Justin P. Hardee, Gordon S. Lynch, Lee B. Miles, Ashley Siegel, Thomas E. Hall, Ralf B. Schittenhelm, Peter D. Currie   +12 more
doaj   +1 more source