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A single-dose gene-editing therapy has shown it can correct the genetic cause of autosomal dominant polycystic kidney disease in preclinical models, slowing cyst growth, improving organ health and potentially leading to future treatments that target the disease at its source.
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.
Japanese researchers have developed a new gene-editing platform, TECHNO, that allows entire human genes – including regulatory regions – to be inserted into mice, allowing for better disease modelling and drug testing.
What can we learn from tumours after they stop responding to treatment? By studying patient tissue directly, researchers are finding new ADC targets that conventional screening often misses.
Researchers have developed a novel CRISPR method that evades the immune system in mice, allowing scientists to study tumour growth and metastasis more accurately.
Researchers have shown that using CRISPR to disable the NRF2 gene can restore chemotherapy sensitivity and slow tumour growth.
Scientists have developed two rapid and affordable blood tests that can detect early markers of Alzheimer’s disease – potentially decades before symptoms appear.
For the first time, researchers have demonstrated that flipping an epigenetic ‘switch’ in memory cells can directly control whether a memory is expressed or silenced, offering new insights into how memories are stored and potentially altered.
CRISPR therapies depend on delivery and lipid nanoparticles are emerging as a more flexible and scalable option than viral vectors.
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.
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
Scientists at Virginia Tech’s Fralin Biomedical Research Institute have identified a promising drug target – PRMT5 – that could make treatment-resistant lung, brain and pancreatic cancers more vulnerable to therapy.
Researchers at the University of Basel and Roche have discovered that slowing the intracellular transport of RNA-based drugs significantly improves their therapeutic impact.
Researchers at Cold Spring Harbor Laboratory have demonstrated that an obscure RNA molecule, LINC01235, plays a crucial role in the progression of aggressive triple-negative breast cancer (TNBC) – potentially allowing for the development of targeted therapies against it.
A new study reveals that blocking the enzyme ST6Gal-I reduces toxic plaque buildup in Alzheimer’s disease by suppressing BACE1 expression - highlighting a new target for future treatment strategies.
