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 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...
CAR-T cell cancer treatments could be made safer and more durable by reprogramming T cells without altering their DNA, according to new research...
CRISPR/Cas9 has been used to identify genome editing targets that significantly enhance the efficiency of a targeted therapy for myeloma, a type of cancer...
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