First clinical trial with in vivo genome editing

Biotech company Sangamo Therapeutics has announced the first in vivo genome editing clinical trial. Patients with haemophilia B, Hurler syndrome or Hunter syndrome will be treated in three separate clinical trials. In all the trials, cuts will be made in the Albumin gene using Zinc-Finger Nucleases after which homologous recombination will integrate a functional copy of the relevant gene into genome. The Albumin gene was chosen as the integration site because it is highly expressed in the liver. In this way, the researchers hope that sufficient therapeutic protein will be produced even if the therapeutic gene only integrates in a minority of liver cells. The treatment consists of intravenous administration of three adeno-associated viruses that contain two genes encoding the Zinc-Finger Nuclease and one gene encoding the therapeutic gene. While ex vivo genome editing has been done before, this will mark the first time where genome editing machinery will edit the genome in cells within the patient’s body. 

For more information on the trials, pleas click here