Gene surgery : repairing the gene

Gene surgery involves repairing the gene itself. Exon skipping, stop codon read-through, meganucleases: these techniques are less than ten years old but already undergoing human trials!

Illustration for article: What is gene surgery?

What is gene surgery?

Gene surgery involves "repairing" defective parts of the gene. It is used during the different stages leading from gene to protein. Depending on the type of gene anomaly, different techniques are considered. Developed less than ten years ago, these very advanced gene surgery techniques - exon skipping, stop codon read-through, surgery using meganucleases, etc. - are already undergoing human trials.
Illustration for article: First advances in gene surgery

First advances in gene surgery

Duchenne muscular dystrophy (DMD) was the subject of the first gene surgery trials. It is a model disease for developing this innovative approach which will have much broader applications to diseases such as cancers, infectious diseases and heart disease.