Making deliberate alterations to the DNA sequence at targeted locations in the genome. This can include deleting, inserting or replacing sections of DNA. Several approaches can be used to achieve these changes including zinc finger nucleases (ZFNs), TALENs, CRISPR/Cas and base editing.
A lipid nanoparticle-based gene therapy for cystic fibrosis restores biological function in a preclinical model...
A topical CRISPR-based genome-editing therapy has been developed to address disease-causing mutations responsible for inherited human skin disorders...
Not all mutations in the CTNNB1 gene — a key cancer driver — are equal, offering insights for personalised cancer treatment...
Professor Jim Al-Khalili interviews Professor George Church in this episode of The Life Scientific. From bringing back mammoths to the Human Genome Project... but does he ask probing enough questions? Davy Tennison finds out for BioNews...
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...
A large-scale study has unveiled genes essential for early brain development and uncovered the cause of a previously unrecognised neurodevelopmental disorder...
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