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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.
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Challenges and opportunities in gene therapy development
Despite the promise of gene therapies, significant challenges have emerged in the field. Dr Carsten Brunn discusses the current obstacles and opportunities when developing gene therapies.
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Novel DNA vaccine for SARS-CoV-2 effective in rodent models
A new DNA vaccine encoding the SARS-CoV-2 Spike protein has shown promise at protecting mice and hamsters against COVID-19.
