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Inhibitory Effects on RNA Binding and RNase H Induction Activity of Prodrug-Type Oligodeoxynucleotides Modified with a Galactosylated Self-Immolative Linker Cleavable by β-Galactosidase.
Bioconjug Chem
October 2024
Department of Life Science and Technology, Tokyo Institute of Technology, Midori-ku, Yokohama, Kanagawa 226-8501, Japan.
Prodrug-type oligonucleotides (prodrug-ONs) are a class of oligonucleotide designed for activation under specific intracellular conditions or external stimuli. Prodrug-ONs can be activated in the target tissues or cells, thereby reducing the risk of adverse effects. In this study, we synthesized prodrug-type oligodeoxynucleotides activated by β-galactosidase, an enzyme that is overexpressed in cancer and senescent cells.
View Article and Find Full Text PDFSafety and efficacy of deoxycytidine/deoxythymidine combination therapy in POLG-related disorders: 6-month interim results of an open-label, single arm, phase 2 trial.
EClinicalMedicine
August 2024
Division of Neurology, Department of Pediatrics, Montreal Children's Hospital, McGill University Health Centre, 1001 Décarie Boulevard, Montreal, Quebec, H4A 3J1, Canada.
Background: DNA polymerase gamma (POLG)-related disorders are a group of rare neurodegenerative mitochondrial diseases caused by pathogenic variants in , the gene encoding POLG. Patients may experience a range of signs and symptoms, including seizures, vision loss, myopathy, neuropathy, developmental impairment or regression, and liver failure. The diseases follow a progressive, degenerative course, with most affected individuals dying within 3 months-12 years of diagnosis.
View Article and Find Full Text PDFConstruction of an autofluorescence interference-free phosphorescence biosensor for the specific detection of TK1 mRNA.
Anal Chim Acta
May 2024
Key Laboratory for Green Organic Synthesis and Application of Hunan Province, Key Laboratory of Environmentally Friendly Chemistry and Application of Ministry of Education, College of Chemistry, Xiangtan University, Xiangtan, 411105, PR China. Electronic address:
The anti-interference ability of biosensors is critical for detection in biological samples. Fluorescence-based sensors are subject to interference from self-luminescent substances in biological matrices. Therefore, phosphorescent sensors stand out among biosensors due to their lack of self-luminescence background.
View Article and Find Full Text PDFTherapeutic targeting at genome mutations of liver cancer by the insertion of HSV1 thymidine kinase through Cas9-mediated editing.
Hepatol Commun
April 2024
Department of Pathology, University of Pittsburg School of Medicine, Pittsburgh, Pennsylvania, USA.
Background: Liver cancer is one of the most lethal malignancies for humans. The treatment options for advanced-stage liver cancer remain limited. A new treatment is urgently needed to reduce the mortality of the disease.
View Article and Find Full Text PDFUnique Attributes of Guinea Pigs as New Models to Study Ocular Herpes Pathophysiology and Recurrence.
Invest Ophthalmol Vis Sci
November 2023
Department of Ophthalmology and Visual Sciences, University of Illinois Chicago, Chicago, Illinois, United States.
Purpose: The objective of this study was to explore the ocular and systemic outcomes of herpes simplex virus type 1 (HSV-1) infection in guinea pigs, to monitor the spontaneous reactivation of the virus, and to assess the effectiveness of various treatments, drawing comparisons to conventional rabbit models.
Methods: Guinea pigs and rabbits were infected in the right corneas with differing doses and strains of HSV-1. Observations were made over a 71-day period, focusing on comparing ocular lesions, viral shedding patterns, and weight loss between the two animal models.
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