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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 PDFASO Visual Abstract: The Impact of Perioperative Radiotherapy on Disease-Specific Survival in Patients with Localized Retroperitoneal Liposarcoma-A Population-Based Propensity-Score Matched Analysis.
Ann Surg Oncol
January 2025
Department of Surgery, Clarunis St. Clara Hospital & University Hospital Basel, Basel, Switzerland.
Characterizing Antisense Oligonucleotide-Induced Histopathology Findings in Spinal Cord of Mauritius Cynomolgus Monkeys by Molecular Localization Investigation.
Toxicol Pathol
January 2025
Novartis Institute for Biomedical Research, Cambridge, Massachusetts, USA.
The safety of a 2'--methoxyethyl antisense oligonucleotide (ASO) was investigated in Mauritius cynomolgus monkeys in a 41-week Good Laboratory Practice (GLP) toxicity study after multiple intrathecal (IT) administrations. Histopathological examination revealed ectopic formation of lymphoid follicles in the spinal cord (SC) at the injection site at all doses and the presence of granular material in neurons of the SC in high-dose animals. The granular material was seen in all the segments of the SC, but mainly in the lumbar segment and persisted at the end of the 26-week recovery period, while the lymphoid follicles showed a reversibility trend.
View Article and Find Full Text PDFASO Author Reflections: Real-World Results of the KEYNOTE-522 Regimen to Guide Surgical Decision Making.
Ann Surg Oncol
January 2025
Department of Breast Surgery, Cleveland Clinic, Lerner Research Institute, Cleveland, OH, USA.
Raman spectra of pyromorphite, vanadinite and mimetite at high pressures and high temperatures.
Spectrochim Acta A Mol Biomol Spectrosc
January 2025
Key Laboratory of High-temperature and High-pressure Study of the Earth's Interior, Institute of Geochemistry, Chinese Academy of Sciences, Guiyang 550081 Guizhou, China. Electronic address:
High-pressure and high-temperature Raman spectra of natural pyromorphite, vanadinite and mimetite were measured up to 11 GPa and 973 K, respectively. No phase transition was observed within the temperature and pressure ranges in this study. Raman modes for pyromorphite, vanadinite and mimetite vary with temperature or pressure linearly.
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