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Getting “under the skin” of frontotemporal dementia
Researchers at the University of East Finland have been using skin cells to investigate pathological hallmarks in frontotemporal dementia patients.
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New skin cell mechanism discovery could treat muscle-related diseases
Scientists have identified the mechanism behind the conversion of skin cells into immature muscle cells, which could lead to therapies preventing muscle degeneration.
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“Noisy” gene expression may help improve stem cell therapies
Researchers have identified a process that amplifies changes in gene expression, which could be harnessed to accelerate stem cell differentiation.
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Inhibiting REST gene could be clue to new diabetes treatments
A study has shown that inhibiting the REST gene boosted insulin-producing cells during early pancreas development in animals.
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Prime editing method corrects cystic fibrosis in stem cells
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
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Molecular insights into LSCC may lead to new drug targets
New insights into lung squamous cell carcinoma (LSCC) have emerged from a proteogenomic study, leading to the identification of potential drug targets.
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Spatial genomic and transcriptomic imaging in drug discovery
In this interview, read about the compilation of a new high-resolution cell atlas of the mouse brain using spatial genomics and transcriptomics.
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Therapeutic target for aggressive cancers discovered through CRISPR
The RNA-modifying protein METTL1 could be targeted to treat some types of aggressive cancers, including brain, blood and kidney.
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Sunlight shown to trigger DNA mutation that causes melanoma
New research found mutations that cause melanoma result from a chemical conversion in DNA fuelled by sunlight, undermining previous theories.
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Prenatal editing halts progression of Hurler syndrome in pre-clinical model
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
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Protein activation discovery could widen access to cancer immunotherapy
The activation of the protein p53 was shown to boost immune responses against cancer tumours in mice in a new study, potentially widening access to immunotherapy.
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Application note: Improving and simplifying the design of knock-in experiments using the TrueDesign Genome Editor
Genome editing technologies have given investigators the power to unlock a variety of new applications and experimental approaches. While gene knockouts have been achievable for several years, successful gene knock-ins have remained elusive due to the low efficiency of homology-directed repair (HDR).
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Human cells can write RNA sequences into DNA, study shows
For the first time, scientists have found evidence that polymerase theta can write RNA segments back into DNA.
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Study provides clues to cause and possible treatment of Parkinson’s
Researchers have shown that the leakage of mitochondrial double-stranded DNA into the cell can contribute to Parkinson's disease.
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Nanoparticle platform enables enhanced delivery of gene therapies
Scientists have created new nanoparticle-based materials that could be used to deliver gene therapies in an adaptable way.