Asthma is among the most common chronic disorders of airways, which affects both children and adults. Asthma being a common disease among different segments of population, it has a high mortality rate and, in the absence of appropriate care, affects the quality of life and leads to economics losses. In a view of continuing growth in the incidence of asthma, it is important to find relevant biological targets for developing new approaches to astma therapy. Recent advances in molecular immunology, genetics, and bioinformatics allowed genes involved in the pathogenesis of asthma to be identified, which provided prerequisites for the development of new types of drugs that can regulate the activity of pathogenically significant genes. To date, a number of technologies for sequence-specific gene regulation (ASO, ribozymes, DNAzymes, EGS, DNA-decoys, U 1-adapters) are available, but RNA interference is the most promising approach in both terms of efficacy and financial cost. This review focuses on the generalization and analysis of experimental data regarding the use of RNA interference technology for the treatment of astma.
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Biol Open
December 2024
Department of Dermatology, University of Zurich, University Hospital Zurich, Schlieren CH-8952, Switzerland.
The gonadal anchor cell (AC) is an essential organizer for the development of the egg-laying organ in the C. elegans hermaphrodite. Recent work has investigated the mechanisms that control the quiescent state the AC adopts while fulfilling its functions.
View Article and Find Full Text PDFAlzheimers Dement
December 2024
University of Pennsylvania, Philadelphia, PA, USA.
Background: Recent genome-wide association studies (GWAS) of Alzheimer's disease (AD) have identified approximately 70 genetic loci linked to the disorder. The pivotal challenge in the post-GWAS era is dissecting the underlying causal variants and effector genes, a crucial step for effective therapeutic development. Most of these variants reside in non-coding regions of the genome, suggesting their regulatory role in distal gene expression.
View Article and Find Full Text PDFAlzheimers Dement
December 2024
University College London, London, United Kingdom.
Background: Mivelsiran (ALN-APP) is an investigational, intrathecally administered RNA interference therapeutic designed to lower levels of amyloid-β (Aβ) peptide, a key driver of Alzheimer's disease (AD) and cerebral amyloid angiopathy (CAA) pathogenesis, by reducing upstream production of amyloid precursor protein (APP). We report additional safety, pharmacodynamic, and biomarker data from the double-blind, placebo-controlled, single ascending dose part of the ongoing mivelsiran Phase 1 study (NCT05231785).
Method: Patients with early-onset AD (symptom onset <65 years of age, Clinical Dementia Rating global score 0.
Int J Med Sci
January 2025
Department of Gastrointestinal Surgery, the Third Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
Chemotherapy resistance is a great challenge in the treatment of gastric cancer (GC), so it is urgent to explore the prognostic markers of chemoresistance. PUF60 (Poly (U)-binding splicing factor 60) is a nucleic acid-binding protein that has been shown to regulate transcription and link to tumorigenesis in various cancers. However, its biological role and function in chemotherapy resistance of GC is unclear.
View Article and Find Full Text PDFJ Saudi Heart Assoc
December 2024
College of Medicine, University of Sharjah, Sharjah, United Arab Emirates.
Objectives: Zilebesiran is an investigational RNA interference therapeutic designed to lower blood pressure by targeting the hepatic production of angiotensinogen, the most upstream precursor of the renin-angiotensin-aldosterone system. This approach aims to offer long-lasting blood pressure control with potentially fewer doses compared to traditional antihypertensive medications. The objective of this systematic review and meta-analysis was to assess the antihypertensive efficacy of zilebesiran in patients with hypertension.
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