Are Zinc Finger Nucleases Making a Comeback?
December 4, 2017
(Genetic Engineering & Biotechnology News) – A few weeks ago, the first person ever had his genes edited from within to treat a genetic disorder. His veins were infused with the investigational genome editing therapy SB-913 to treat his disease, mucopolysaccharidosis type II (MPS II). MPS II, often called Hunter syndrome, is a rare genetic disorder caused by a mutation in the iduronate 2-sulfatase (IDS) gene and occurs almost exclusively among males. The therapy uses the gene-editing tool called zinc finger nucleases to cut out the IDS mutated gene and replace it with the healthy gene. The patient received the therapy as part of a Phase I/II clinical trial.