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Adenine base editor corrected ADPKD point mutations in hiPSCs and kidney organoids.
Adv Biotechnol (Singap)
June 2024
MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory of Biocontrol, School of Life Sciences, Sun Yat-Sen University, Guangzhou, Guangdong, 510275, China.
Autosomal dominant polycystic kidney disease (ADPKD) is a dominant genetic disorder caused primarily by mutations in the PKD1 gene, resulting in the formation of numerous cysts and eventually kidney failure. However, there are currently no gene therapy studies aimed at correcting PKD1 gene mutations. In this study, we identified two mutation sites associated with ADPKD, c.
View Article and Find Full Text PDFEvaluation of Mesoporous Silica Nanoparticles as Carriers of Triarylmethyl Radical Spin Probes for EPR Oximetry.
J Phys Chem B
January 2025
Department of Pharmaceutical Sciences, School of Pharmacy, West Virginia University, Morgantown, West Virginia 26506, United States.
measurement and mapping of oxygen levels within the tissues are crucial in understanding the physiopathological processes of numerous diseases, such as cancer, diabetes, or peripheral vascular diseases. Electron paramagnetic resonance (EPR) associated with biocompatible exogenous spin probes, such as Ox071 triarylmethyl (TAM) radical, is becoming the new gold standard for oxygen mapping in preclinical settings. However, these probes do not show tissue selectivity when injected systemically, and they are not cell permeable, reporting oxygen from the extracellular compartment only.
View Article and Find Full Text PDFRetinoid-impregnated nanoparticles enable control of bone growth by site-specific modulation of endochondral ossification in mice.
J Bone Miner Res
January 2025
Department of Orthopaedics, University of Maryland School of Medicine, Baltimore, MD.
Growth-plate (GP) injures in limbs and other sites can impair GP function and cause deceleration of bone growth, leading to progressive bone lengthening imbalance, deformities and/or physical discomfort, decreased motion and pain. At present, surgical interventions are the only means available to correct these conditions by suppressing the GP activity in the unaffected limb and/or other bones in the ipsilateral region. Here, we aimed to develop a pharmacologic treatment of GP growth imbalance that involves local application of nanoparticles-based controlled release of a selective retinoic acid nuclear receptor gamma (RARγ) agonist drug.
View Article and Find Full Text PDFCharacterizing 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 PDFRecurrence of skin and soft tissue infections: identifying risk factors and treatment strategies.
Curr Opin Infect Dis
January 2025
Department of Medical and Surgical Sciences, Alma Mater Studiorum University of Bologna.
Purpose Of Review: Recurrent skin and soft tissue infections (RSSTIs) are challenging for the clinicians due to morbidity and healthcare-related costs. Here, we review updates on risk factors and management.
Recent Findings: RSSTIs rates range between 7 and 45%.
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