A multicentre trial has demonstrated that patients receiving stem cell transplants from unrelated donors with multiple genetic mismatches can achieve survival rates comparable to those with better-matched donors when using post-transplant cyclophosphamide prophylaxis.
Researchers have developed a third-generation CRISPR technique that modifies gene expression by removing methylation marks rather than cutting DNA sequences. The approach uses catalytically dead Cas9 proteins fused to demethylating enzymes, offering a potentially safer alternative to conventional gene editing for treating inherited blood disorders including sickle cell disease and β-thalassemia.
Mayo Clinic researchers have developed BACDAC, a computational tool that exposes elusive genomic patterns in cancer samples with low DNA coverage, with the aim of transforming how clinicians predict tumour behaviour and guide personalised treatment decisions through enhanced detection of chromosomal instability markers.
A groundbreaking stem cell repository has been established using blood samples from over 100 individuals selected for their extreme genetic risk profiles for Alzheimer’s disease. The IPMAR resource will enable researchers worldwide to study how genetic variants contribute to the neurodegenerative condition in laboratory models.
A major NHS study has demonstrated that pharmacogenomic testing can be successfully integrated into routine clinical practice, with genomic data seamlessly incorporated into electronic health records to guide safer prescribing decisions. The PROGRESS programme showed that 95% of patients carried actionable genetic variants related to common medications, leading to prescription adjustments in over a quarter […]
A pioneering five-year research programme to develop the foundational tools for synthesising human genomes has launched with £10mn funding from Wellcome. The ambitious Synthetic Human Genome project, led by Professor Jason Chin at the MRC Laboratory of Molecular Biology, aims to unlock transformative biotechnology applications and accelerate targeted cell-based therapies.
Scientists at the San Raffaele Telethon Institute for Gene Therapy have unlocked a remarkable biological window – a fleeting period immediately after birth when circulating blood stem cells become uniquely accessible for genetic modification directly within the body. This discovery could transform treatment approaches for devastating genetic blood disorders, offering hope without the invasive procedures […]
Researchers at Children’s Hospital of Philadelphia and Penn Medicine have successfully treated an infant with a rare genetic disease using a bespoke CRISPR gene editing therapy, marking a watershed moment in personalised genomic medicine.
Researchers have developed a novel RNA-based gene editing system capable of inserting entire therapeutic genes, addressing limitations of current CRISPR technology for diseases with multiple mutations.
University of Chicago researchers have developed a groundbreaking imaging technology that visualizes the spatial organization of genetic material in three dimensions, without relying on conventional optical instruments. This technique, called volumetric DNA microscopy, works by tagging DNA and RNA molecules and allowing neighboring tags to interact, creating a molecular network that encodes their relative positions […]
April 2024
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callan emery / Midjourney
callan emery / Midjourney
Children’s Hospital of Philadelphia