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[Antisense oligonucleotide as novel therapies for neurogenetic disorders].
Zhonghua Yi Xue Yi Chuan Xue Za Zhi
January 2025
University of Hong Kong Shenzhen Hospital, Shenzhen, Guangdong 518053, China.
Antisense oligonucleotide (ASO) was discovered several decades ago and initially used only as a research tool in the laboratory. In recent years, several ASO therapeutics have been developed for neurological disorders. Some of these therapeutics, including eteplirsen, golodirsen, viltolarsen, nusinersen and inotersen, have been approved by the Food and Drug Administration (FDA) and begun to draw the public's attention as an effective therapeutic approach.
View Article and Find Full Text PDFRandomized, observer-blind, controlled Phase 1 study of the safety and immunogenicity of the Na-GST-1/Alhydrogel hookworm vaccine with or without a CpG ODN adjuvant in hookworm-naïve adults.
PLoS Negl Trop Dis
December 2024
Department of Microbiology, Immunology and Tropical Medicine, School of Medicine and Health Sciences, The George Washington University, Washington, DC, United States of America.
Background: Recombinant Necator americanus Glutathione-S-Transferase-1 (Na-GST-1) formulated on Alhydrogel (Na-GST-1/Alhydrogel) is being developed to prevent anemia and other complications of N. americanus infection. Antibodies induced by vaccination with recombinant Na-GST-1 are hypothesized to interfere with the blood digestion pathway of adult hookworms in the host.
View Article and Find Full Text PDFNanotechnology-Based Approaches for the Management of Diabetes Mellitus: An Innovative Solution to Long-Lasting Challenges in Antidiabetic Drug Delivery.
Pharmaceutics
December 2024
Department of Obstetrics and Gynecology, Grigore T. Popa University of Medicine and Pharmacy, 700111 Iasi, Romania.
Diabetes is a widespread metabolic illness. Mismanagement of diabetes can lead to severe complications that tremendously impact patients' quality of life. The assimilation of nanotechnology in diabetes care holds the potential to revolutionize treatment paradigms, improve patient outcomes, and reduce the economic burden associated with this pervasive disease.
View Article and Find Full Text PDFBichromatic Splicing Detector Allows Quantification of and Splicing Isoforms in Single Cells by Fluorescent Live-Cell Imaging.
Int J Mol Sci
December 2024
Department of Neuropediatrics, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität Berlin, and Berlin Institute of Health, 13353 Berlin, Germany.
Thyroid hormone receptor alpha (THR) is a nuclear hormone receptor that binds triiodothyronine (T3) and acts as an important transcription factor in development, metabolism, and reproduction. The coding gene, , has two major splicing isoforms in mammals, and , which encode THR1 and THR1, respectively. The better characterized isoform, THR1, is a transcriptional stimulator of genes involved in cell metabolism and growth.
View Article and Find Full Text PDFAn Approach to Identifying Single-Nucleotide Mutations Using Noncovalent Associates of Gold Nanoparticles with Fluorescently Labeled Oligonucleotides.
Int J Mol Sci
December 2024
Institute of Chemical Biology and Fundamental Medicine SB RAS, Novosibirsk 630090, Russia.
Globally, widespread tuberculosis is one of the acute problems of healthcare. Drug-resistant forms of tuberculosis require a personalized approach to treatment. Currently, rapid methods for detecting drug resistance of (MTB) to some antituberculosis drugs are often used and involve optical, electrochemical, or PCR-based assays.
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