All RNAs articles
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ArticleTurning GWAS signals into drug targets with scalable CRISPR
Genome-wide association studies have linked thousands of genetic variants to disease, yet most remain disconnected from drug-relevant biology. Neville Sanjana, Professor at New York University and Core Faculty Member at the New York Genome Center, explains how scalable CRISPR screens systematically link noncoding variants to causal genes and therapeutic targets.
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NewsPERM1 protein linked to heart recovery in LVAD patients
A newly identified protein may explain why some failing hearts recover function following mechanical support while others do not.
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NewsCircio’s circular RNA platform shows 40-fold gene expression boost
Circio Holding has presented preclinical data at ASGCT demonstrating that its circVec circular RNA platform achieved up to 40-fold greater gene expression in cardiac tissue and 50-fold in ocular tissue compared with conventional AAV gene therapy approaches, potentially enabling significant dose reductions and improved safety profiles.
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NewsNew RNA sequencing method reveals molecular structures at single-molecule resolution
A*STAR scientists have developed sm-PORE-cupine, a nanopore-based sequencing method that maps individual RNA structures at single-molecule resolution.
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NewsCells use nanoparticle couriers to exchange biological information
University College Dublin researchers have discovered that cells use nanoparticle-based courier systems coated with proteins and RNA to exchange biological information. The findings detail how natural cellular gateways could be exploited to deliver therapeutic molecules to previously inaccessible locations, potentially changing the way RNA, gene and protein-based therapies work.
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NewsNew platform reveals shared drug targets across genetic mutations in cancer and neurodegeneration
Researchers have developed PerturbFate, a platform that simultaneously tracks gene expression, RNA dynamics and chromatin accessibility across thousands of genetic perturbations in single cells. Applied to melanoma drug resistance, the technology revealed that diverse mutations converge on shared regulatory nodes, offering a route to therapies targeting common mechanisms rather than individual genetic alterations.
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NewsNasal spray reverses age-related brain inflammation in preclinical study
Researchers at Texas A&M University have developed a nasal spray delivering extracellular vesicles that reverses neuroinflammaging in preclinical models. Two doses significantly reduced brain inflammation, restored mitochondrial function and improved memory within weeks, with effects persisting for months. The therapy bypasses the blood-brain barrier and targets inflammatory pathways whilst reactivating cellular energy production.
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NewsCircio and Acuitas partner on circular RNA CAR T therapy
Norway-based biotechnology company Circio Holding ASA has partnered with Acuitas Therapeutics to evaluate its circVec circular RNA platform for in vivo CAR T cell therapy. The collaboration will combine Circio’s technology, which reportedly delivers gene expression lasting up to six months in lymphocytes, with Acuitas’ lipid nanoparticle delivery system targeting specific T cell populations for potential applications in oncology and autoimmune diseases.
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NewsLactylation emerges as key driver of lung cancer resistance
New research from Shanghai Pulmonary Hospital outlines how lactate-driven lactylation acts as a metabolic switch controlling epigenetic regulation in lung cancer. The findings reveal self-reinforcing feedback loops that sustain drug resistance and suggest novel therapeutic strategies targeting the enzymes and pathways that maintain this process.
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NewsLipid nanoparticles deliver dual therapy for lung cancer
Oregon State University researchers have developed engineered lipid nanoparticles that deliver follistatin mRNA directly to lung tumours, simultaneously targeting cancer growth and muscle-wasting cachexia in preclinical studies.
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NewsDNA ‘needle’ bypasses cellular defences to deliver therapeutics
A microscopic DNA nanostructure inspired by bacteriophages can deliver therapeutic molecules directly into cells whilst evading endosomal traps that typically render treatments ineffective. The virus-inspired platform, demonstrated in breast cancer cells, could address the longstanding challenge that only one per cent of oligonucleotide therapeutics reach their cellular targets.
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ReportCRISPR & Genomics: Turning Data into Confident Drug Discovery Decisions
Early drug discovery has no shortage of genomic data, but confidence remains scarce. This report examines how CRISPR, functional genomics and human-relevant models are being applied to determine which signals matter, how they influence disease biology and which targets and strategies are worth pursuing.
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NewsNew RNA injection could help hearts heal after heart attacks
A new RNA-based therapy developed at Columbia University could help the heart repair itself after a heart attack without invasive surgery.
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NewsSimple amino acid mix dramatically improves gene therapy delivery
Scientists have discovered that adding a simple cocktail of three common amino acids to lipid nanoparticle injections can dramatically improve the delivery of mRNA and CRISPR therapies.
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NewsLab-made lncRNA could lead to new anti-inflammatory drugs
Scientists at the University of Toronto have created long noncoding RNA outside living cells for the first time, a breakthrough that has already produced experimental anti-inflammatory molecules.
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NewsCircular RNA drives 50-fold boost in eye gene therapy
New in vivo data show a circular RNA platform can deliver up to a 50-fold increase in gene expression in the eye compared with conventional mRNA-based AAV approaches.
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NewsMessy lipid nanoparticles deliver RNA more effectively
New research shows that lipid nanoparticles used in mRNA vaccines may deliver RNA more effectively when their structure is disorganised.
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NewsVirus-mimicking LENN nanoparticles deliver mRNA to bladder cancer cells
Researchers have developed virus-inspired LENN nanoparticles that deliver mRNA therapies directly to bladder cancer cells, remain stable after freeze-drying and avoid triggering immune responses.
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Newsinsitro expands AI drug discovery with CombinAbleAI acquisition
insitro has acquired Israeli AI therapeutics company CombinAbleAI and launched its TherML platform, creating an end-to-end, modality-agnostic system for designing small molecules, antibodies, oligonucleotides and other complex biologics.
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NewsHow aggressive breast cancer evades the immune system
Researchers in China have discovered a previously unknown mechanism that allows aggressive breast cancers to avoid immune attack, while simultaneously exposing a weakness that could make these tumours especially responsive to existing immunotherapy treatments.