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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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CRISPR
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Application note: Effectiveness of CRISPR‑Cas9 using pools of synthetic crRNAs in high-content analysis screening experiments
Chemical synthesis of guide RNAs for CRISPR-Cas9 gene editing enables accurate and rapid production of CRISPR libraries and screening in an arrayed, one-gene-per-well fashion.
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New algorithm could make CRISPR gene editing more precise
Researchers have created an algorithm that can predict the efficiencies of guide RNAs for CRISPR, potentially making it more precise.
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Novel CRISPR strategy developed for Duchenne muscular dystrophy
A CRISPR gene editing technique has been developed to restore dystrophin, which is missing in many Duchenne muscular dystrophy (DMD) patients.
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CRISPR reveals genes involved in zebrafish spinal cord regeneration
A CRISPR-Cas9 method has been used to identify which genes play a role in repairing zebrafish spinal cord injuries.
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Scientists call for awareness of unintended mutations from CRISPR
Scientists have said that researchers need to be more aware of unintended mutations to human embryos following CRISPR-Cas9 genome editing.
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Scientists develop new CRISPR-based tool to control epigenome
Researchers have used CRISPR to create a new technology that can switch off almost any gene in cells, called CRISPRoff.
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Application note: Effectiveness of CRISPR‑Cas9 using pools of synthetic crRNAs in high-content analysis screening experiments
In this application note, discover how pools of synthetic crRNAs can produce a phenotype as strong as, or stronger, than any one of the component crRNAs in arrayed CRISPR screening experiments.
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Prime editing demonstrates success at precise gene editing in mice
Researchers have shown that prime editing is able to effectively edit genes in mice but without the off-target effects of CRISPR.
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Nano-micelles used in CRISPR genome editing of mouse brains
Researchers have reported that nano-micelles can be used to efficiently deliver CRISPR-Cas9 to edit genes in the brains of mice.
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CRISPR-dead Cas9 could offer non-addictive treatment for pain, study shows
A new CRISPR gene therapy for chronic pain has been shown in mice to temporarily repress a gene involved in sensing pain.
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Nanobodies could help CRISPR turn genes on and off
Researchers have used nanobodies to act as an assistant to CRISPR, bringing in effectors to turn specific genes on and off.
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New CRISPR tool could enable gene editing over time, study shows
Researchers have shown that a guide RNA can be used in CRISPR gene editing to ensure sequential Cas9 cuts to DNA.
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CRISPR used to model acute myeloid leukaemia progression
CRISPR-Cas9 and stem cell technologies have been used to create a cellular model of acute myeloid leukaemia, revealing therapeutic targets.
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TALEN is a more efficient gene editing tool for tightly packed DNA, finds study
TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.
