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Highlights from Drug Target Review’s Cell & Gene Therapy Advancements Online Summit
Here, we round up some of the key takeaways from the expert panel discussions at the Cell & Gene Therapy Advancements Online Summit.
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CRISPR
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Improving the safety of CRISPR for the clinic
Dr Danilo Maddalo from Genentech outlines the main benefits of CRISPR within the clinical realm and highlights some of the safety concerns that must be considered before CRISPR products reach patients.
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Groundbreaking gene-editing method creates single-sex mice litters
The exciting new study demonstrates how the technology could be used to improve animal welfare in scientific research and agriculture.
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Positive pre-clinical results for CRISPR-based HIV therapy
The CRISPR-based therapy called EBT-101 excised HIV proviral DNA from the genomes of different cells and tissues in human cells and mice.
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Researchers discover new link between CRISPR, p53 and cancer
The new findings suggest that inhibiting p53 can reduce CRISPR-associated cancer risks and may lead to developments in precision medicine.
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CRISPR screen unveils enzyme target for inflammatory diseases
Scientists used a CRISPR-based screen to find that inhibiting MTHFD2 reduces disease severity in inflammatory disease models.
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New approach improves avoidance of ‘bystander’ edits in CRISPR-base editor treatments
Rice University scientists have refined specific CRISPR-base editing strategies to avoid errors that occur during gene editing.
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Researchers achieve DNA methylation removal in mouse models
The study used CRISPR to show that DNA “de-methylation” activity can be targeted to anywhere in the DNA and may be a new therapeutic strategy.
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Tropical frog models reveal novel kidney disease insights
Scientists utilised CRISPR technology and deep learning systems to investigate the genes associated with polycystic kidney disease.
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Two-week-old human embryos reveal origin of Huntington’s disease
Scientists have discovered a signalling pathway alteration in embryos with Huntington’s disease, paving the way for ground-breaking treatments.
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CRISPR screening tool exposes new leukaemia drug target
A CRISPR screening tool identified a new target for acute myeloid leukaemia with fewer side effects than current approaches.
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Scientists uncover mechanisms of potential COVID-19 drug targets
Researchers have used CRISPR and cryogenic electron microscopy to unravel the workings of two receptors involved in diseases such as cancer and COVID-19.
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CRISPR-based droplet method speeds up gene therapy research
The new CRISPR-based technology called MIC-Drop rapidly identified several genes for heart development and function in zebrafish.
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CRISPR-Cas9-based gene dropout screens: a powerful platform for drug discovery
CRISPR holds great promise in advancing pharmacological research and has fuelled the rapid expansion of using gene-edited cells for drug discovery processes. CRISPR-Cas9 dropout screens have emerged as a useful tool for high-throughput large-scale loss-of-function screens, which seek to identify the relationship between genotype and phenotype. Dr Pushpanathan Muthuirulan, Research…
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Application note: Streamlined genome engineering in picodroplets
A picodroplet-based microfluidic method for automating the complete genome-editing workflow. Learn more in this free application note.
