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http://dx.doi.org/10.1038/mt.2016.47 | DOI Listing |
Invest Ophthalmol Vis Sci
September 2025
Department of Ophthalmology, Dean McGee Eye Institute, University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States.
Purpose: Adeno-associated viruses (AAVs) have become the preferred vector for gene therapy in ophthalmology. However, requirements for specific cell surface receptors limit AAV-mediated retinal cell transduction efficiency. This led to the need to engineer novel AAV vectors for widespread retinal transduction and transgene expression.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
September 2025
Neuroscience Research Australia, Sydney, NSW 2031, Australia.
Optogenetics offers a minimally invasive, low-fatigue, and temporally precise alternative to electrical stimulation for skeletal muscle control. After opsin expression in muscle cells, contraction can be stimulated with light. Obstructive sleep apnea, characterized by repeated airway collapse during sleep, suits this approach, as upper airway muscles are readily accessible via the oral cavity, and require stimulation synchronized to respiration.
View Article and Find Full Text PDFRedox Biol
September 2025
College of Veterinary Medicine and BK21 FOUR Program, Chonnam National University, Gwangju, 61186, Republic of Korea. Electronic address:
Copper oxide nanoparticles (CuONPs) are increasingly used across various industrial applications, raising concerns about their potential toxicity and necessitating comprehensive safety evaluations. In this study, we first evaluated the respiratory toxicity of CuONP exposure in a mouse model of asthma. CuONP exposure alone exacerbated asthma symptoms, as evidenced by increased airway hyperresponsiveness, inflammatory cell infiltration, and elevated cytokine production with increasing thioredoxin-interacting protein (TXNIP) expression.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
September 2025
Department of Pediatrics, UT Southwestern Medical Center, Dallas, TX 75390, USA.
Surfeit locus protein 1 (SURF1)-related Leigh syndrome is an early-onset neurodegenerative disorder characterized by a reduction in complex IV activity that disrupts mitochondrial function. Currently, there are no disease-modifying treatments available. Previously, we reported that a gene replacement therapy for -related Leigh syndrome was developed, which showed improved complex IV activity and restored exercise-induced lactate acidosis, as well as a high safety profile in wild-type (WT) mice.
View Article and Find Full Text PDFCancer Lett
August 2025
Department of Hepatobiliary and Pancreatic Surgery, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Pancreatic Disease, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Institute of T
Bioinformatics-based association study revealed a strong positive correlation between UBE2C, an E2 ubiquitin-conjugating enzyme, and pancreatic cancer and patient survival. However, whether and how UBE2C plays a causal role in pancreatic tumorigenesis remains elusive. Here, we report that UBE2C functions as a promoter in this process.
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