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Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.
J Muscle Res Cell Motil
January 2025
Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Dr, Headington, Oxford, OX3 7TY, UK.
Recent years have seen enormous progress in the field of advanced therapeutics for the progressive muscle wasting disease Duchenne muscular dystrophy (DMD). In particular, four antisense oligonucleotide (ASO) therapies targeting various DMD-causing mutations have achieved FDA approval, marking major milestones in the treatment of this disease. These compounds are designed to induce alternative splicing events that restore the translation reading frame of the dystrophin gene, leading to the generation of internally-deleted, but mostly functional, pseudodystrophin proteins with the potential to compensate for the genetic loss of dystrophin.
View Article and Find Full Text PDFAlpelisib is a phosphatidylinositol 3-kinase inhibitor approved by the US Food and Drug Administration for the treatment of hormone receptor-positive metastatic breast cancer with (phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit α) mutation. In recent years a number of adverse effects have been observed to be associated with this therapy, the most notable of which is hyperglycemia. A literature search was conducted to include case studies, case series, systematic reviews, and meta-analyses within the last 10 years that evaluated patients with mutated hormone receptor-positive, human epidermal growth factor receptor 2 negative metastatic breast cancer.
View Article and Find Full Text PDFThe field of π-conjugated organic materials has seen significant advances in recent years. However, enhancing the functionality of well-established, mass-produced compounds remains a considerable challenge, despite being an intriguing strategy for designing high-value organic materials with low production costs. In this context, vat dyes, known for their wide range of colors and extensive use in the textile industry are particularly attractive.
View Article and Find Full Text PDFExplorative Study of Modulatory Effects of Notochordal Cell-Derived Extracellular Vesicles on the IL-1β-Induced Catabolic Cascade in Nucleus Pulposus Cell Pellets and Explants.
JOR Spine
March 2025
Department of Clinical Sciences, Faculty of Veterinary Medicine Utrecht University Utrecht Netherlands.
Background: Cell-free regenerative strategies, such as notochordal cell (NC)-derived extracellular vesicles (EVs), are an attractive alternative in developing new therapies for intervertebral disc (IVD) degeneration. NC-EVs have been reported to elicit matrix anabolic effects on nucleus pulposus cells from degenerated IVDs cultured under basal conditions. However, the degenerative process is exacerbated by pro-inflammatory cytokines contributing to the vicious degenerative cycle.
View Article and Find Full Text PDFAdvancing veterinary vaccines design through trained immunity insights.
Front Vet Sci
January 2025
College of Life Science, Longyan University, Longyan, China.
Trained immunity, characterized by long-term functional reprogramming of innate immune cells, offers promising new directions for veterinary vaccine development. This perspective examines how trained immunity can be integrated into veterinary vaccine design through metabolic reprogramming and epigenetic modifications. We analyze key molecular mechanisms, including the shift to aerobic glycolysis and sustained epigenetic changes, that enable enhanced immune responses.
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