A genome-editing platform that uses an enzyme (Cas9) to make a double-stranded break at a specific location in the genome, directed by a guide RNA.
A new CRISPR-based approach has been designed to selectively eliminate human cells with disease-causing genes in vitro...
The physical shape of DNA in a cell influences the rate of unintended, off-target effects of CRISPR/Cas9 genome editing...
A lipid nanoparticle-based gene therapy for cystic fibrosis restores biological function in a preclinical model...
CRISPR/Cas9 genome editing has been used in mice to investigate two key genes' roles in male fertility and sperm production...
A new genome editing approach has been shown to safely and efficiently remove the source of transthyretin amyloidosis in mice, demonstrating greater therapeutic opportunities for genome editing...
Data on the effectiveness and safety of Casgevy gene therapy in children aged five to 11 years has been reported...
CRISPR-based genome editing has been used to overcome chemotherapy resistance in lung tumours by disabling a tumour-specific mutation...
Two gene therapy trials run by Intellia Therapeutics have been paused after a study participant was hospitalised...
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