All Gene Therapy articles
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InterviewComputational design drives new generation of synthetic promoters
SynGenSys applies computational design strategies to engineer synthetic promoters with predictable performance characteristics for therapeutic and manufacturing applications. Professor David James discusses how tissue-specific regulatory elements are designed from genomic data to enable precise control of gene expression in contexts ranging from NK cell immunotherapy to biologic production.
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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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ArticlePreclinical takeaways from the World ADC Conference London
Experts from the World ADC Conference in London highlight how patient-centric, predictive preclinical tools and innovative ADC designs are improving safety, efficacy and clinical translation.
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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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NewsNew SHANK3 conditional knockout mouse model launched for PMS research
A new SHANK3 conditional knockout mouse model from InnoSer, CureSHANK and Ozgene has been launched to advance research into Phelan-McDermid syndrome, other neurodevelopmental disorders and the development of new therapies.
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NewsELRIG announces keynote speakers for Cell and Gene Therapy 2026 in Cambridge
ELRIG (European Laboratory Research & Innovation Group) has announced the keynote speakers for its inaugural Cell and Gene Therapy 2026 conference, taking place at Hinxton Hall in Cambridge from 9–10 March.
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ArticleThe mother of invention: from steam engines to AI-designed drugs
Every great leap in history started with a single, urgent need. Now AI is emerging as the next great engine of invention, transforming the future of medicine faster than ever imagined.
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NewsNew stealth CRISPR method reduces immune interference in tumours
Researchers have developed a novel CRISPR method that evades the immune system in mice, allowing scientists to study tumour growth and metastasis more accurately.
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NewsCRISPR disruption of NRF2 re-sensitises tumours to chemo
Researchers have shown that using CRISPR to disable the NRF2 gene can restore chemotherapy sensitivity and slow tumour growth.
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ArticleResetting retinal networks with a gene-agnostic approach
What if treating blindness did not mean fixing a single faulty gene? Find out how modifier gene therapy is designed to protect vision by targeting broader retinal pathways.
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ArticleCircular RNA technology: the future of gene therapy
Pioneering circular RNA could redefine what the future of gene therapy looks like. Erik Digman Wiklund, CEO of Circio, shares how his company’s platform is enhancing gene expression and tackling toxicity challenges through smarter design and scientific collaboration.
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NewsNew mRNA therapy could inform future male infertility treatments
Scientists have restored sperm production in mice with a genetic form of male infertility using mRNA delivered via lipid nanoparticles, with the hope of informing future treatments for non-obstructive azoospermia.
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ArticleSPNS1 mutations reveal new lysosomal lipid recycling pathway
Scientists have linked rare mutations in SPNS1 to a previously unknown lipid recycling pathway in lysosomes, revealing how faulty fat processing can trigger muscle and liver disease.
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ArticleStreamlining Biomanufacturing of Personalised Cancer Immunotherapies with Synthetic DNA
A Synthetic DNA Approach for Speed, Scale & Flexibility
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NewsNew gene therapy restores brain function in SYNGAP1 disorder
Scientists have developed a new gene therapy that reversed symptoms of SYNGAP1-related brain disorders in mice, which could lead to new treatments for this group of neurological conditions.
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ArticleAdvancing gene editing: the role of lipid nanoparticles in CRISPR delivery
CRISPR therapies depend on delivery and lipid nanoparticles are emerging as a more flexible and scalable option than viral vectors.
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ArticleBetter assays: the key step in moving drugs from lab to clinic
From gene therapy to Long Covid, better assays are helping researchers move promising drug candidates from early studies into clinical trials. Dr Alexandre Lucas explains the technologies, challenges and innovations driving this progress.
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NewsNew nanostructure makes CRISPR edits safer and three times faster
Northwestern University researchers have created DNA-coated nanoparticles that deliver CRISPR into cells three times more effectively while reducing toxicity. The advance could overcome one of the biggest barriers to gene-editing therapies.
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NewsKey newborn liver cells could boost paediatric gene therapy
A small subset of newborn liver cells – known as clonogenic hepatocytes – drives over 90 percent of adult liver growth. New research shows how targeting these cells early could improve the effectiveness and durability of paediatric gene therapies.
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Scientists chart ovarian reserve to help advance new infertility treatments
UCLA scientists have created the first detailed map of how the ovarian reserve forms in primates, offering new insights – and potential new treatments – for infertility, polycystic ovary syndrome (PCOS) and hormone-related conditions.