RNA Interference News and Research

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SHASTA trials show substantial triglyceride reductions with plozasiran

Plozasiran significantly reduced triglyceride levels in severe hypertriglyceridemia patients, showing promise in preventing acute pancreatitis after one year.

31 Aug 2026

Blocking the Axl receptor may offer a new way to promote bone formation

Osteoporosis is one of the most common age-related bone diseases, affecting millions of people worldwide and increasing the risk of fractures, disability, and reduced quality of life.

28 Jul 2026

AI-powered analytics and experimental validation: Transforming target identification in drug discovery

Discover how AI-powered analytics and experimental validation combine to improve target identification and accelerate drug discovery success.

From Axxam S.p.A 23 Jul 2026

Scientists capture atomic images to improve RNA interference drug design

RNA interference is a natural mechanism for living cells to control whether specific genes are being used or not.

29 Jun 2026

Cells detect and silence invading transposons through abnormal RNA signals

Transposons, DNA sequences that can self-replicate and move (jump) throughout the genome, are widespread and can affect cell survival if left unchecked. Cells control these "jumping genes" by silencing them, but little was known about how cells recognize and defend themselves from invading transposons.

26 May 2026

Ohio State study unlocks the long-standing mystery of RNA interference

While scientists have known for over two decades that all cells use a strategy called RNA interference to regulate gene expression, a new study is the first to describe how a specific protein manages the step-by-step process of assembling the molecular complex that performs the regulatory job.

26 May 2026

Twice-yearly blood pressure treatment could reshape hypertension care, but doctors warn against a “fire-and-forget” approach

Long-acting RNA interference therapy for hypertension could shift blood pressure control from daily pill-taking to scheduled, system-led protection. But the authors warn that this “vaccine-like” model must preserve lifestyle support, monitoring, and follow-up to avoid clinical disengagement.

27 Apr 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid deposits of misfolded transthyretin (TTR) proteins.

14 Jan 2026

Fruit fly study identifies genes regulating dopamine and sleep

Dopamine in the brain influences movement, learning, motivation and sleep. In humans, problems with dopamine are linked to conditions like Parkinson's disease, depression and sleep disorders.

13 Jan 2026

Artificial intelligence unlocks new frontiers in RNA drug design

In the realm of modern medicine, RNA-based therapies have emerged as a promising avenue, with significant advancements in metabolic diseases, oncology, and preventive vaccines. A recent article published in Engineering titled "The Future of AI-Driven RNA Drug Development" by Yilin Yan, Tianyu Wu, Honglin Li, Yang Tang, and Feng Qian, explores how artificial intelligence (AI) can revolutionize RNA drug development, addressing current limitations and offering new opportunities for innovation.

31 Dec 2025

Novel biotechnical vector integrates viral and synthetic features for targeted cancer therapy

Gene therapy offers the possibility of addressing cancer at its molecular roots by targeting disease-causing genes rather than relying solely on surgery, chemotherapy, or radiotherapy.

16 Sep 2025

Phase II trial finds zilebesiran lowers systolic blood pressure in patients with uncontrolled hypertension

Among individuals with cardiovascular disease or high cardiovascular risk and uncontrolled hypertension on multiple anti-hypertensive agents, a single subcutaneous dose of zilebesiran 300 mg led to a 5-mmHg reduction in systolic blood pressure at Month 3 compared with placebo, a difference that did not reach statistical significance.

1 Sep 2025

Breakthroughs in diagnosis and therapy for transthyretin amyloidosis

Transthyretin amyloidosis (ATTR) is caused by the misfolding and aggregation of the transthyretin (TTR) protein, leading to multi-organ damage.

21 Aug 2025

'Two-in-one' RNA molecule can simultaneously turn off two difficult-to-target cancer-related genes

University of North Carolina Lineberger Comprehensive Cancer Center researchers have developed a "two-in-one" molecule that can simultaneously turn off two notoriously difficult-to-target cancer-related genes, KRAS and MYC, as well as directly deliver drugs to tumors that express these genes.

4 Aug 2025

Circular RNAs emerge as key players in hepatitis B-related liver cancer

Hepatitis B virus-induced hepatocellular carcinoma (HCC) remains a major global health burden, and new therapeutic and diagnostic approaches are urgently needed.

20 Jun 2025

New injection therapy can lead to long lasting blood pressure reduction

Results from a clinical trial led by researchers from Queen Mary University of London, published today in JAMA, show that giving people with high blood pressure an injection every six months can lead to a meaningful, sustained reduction in their blood pressure.

28 May 2025

Fungal antiviral responses controlled by RNA editing and transcription factor dynamics

The expression of symptoms of viral infections is a byproduct of complex virus-host molecular pathways.

23 May 2025

Exploring the role of RNA binding proteins and circular RNAs in cancer

The interaction between RNA-binding proteins (RBPs) and circular RNAs (circRNAs) has emerged as a key area of interest in understanding cancer biology.

12 May 2025

Influenza virus hijacks human gene regulator to evade immune system

The influenza virus manipulates the body's gene regulation system to accelerate its own spread, according to researchers at the University of Gothenburg.

28 Apr 2025

lncRNAs shape the tumor microenvironment and drive cancer development

The tumor microenvironment (TME) is a complex and dynamic network consisting of tumor cells, immune cells, stromal cells, extracellular matrix (ECM), cytokines, and growth factors, all interacting to influence tumorigenesis, progression, and metastasis.

24 Apr 2025