Fig. 3
From: Alpha-1 antitrypsin deficiency: outstanding questions and future directions
Strategies for delivery of engineered nucleases. a Cell-based (ex-vivo) approach. The therapeutic engineered nucleases are packaged into a delivery vehicle (virus, liposomes, naked-DNA, etc). Cells from patient carrying the mutated non-functioning gene are isolated and transfected with engineered nucleases to correct the mutated gene. Modified “healthy” cells are expanded in vitro and test for safety and off-target effects before being re-administered to the patient. b Direct-delivery (in vivo) approach. In that case, the therapeutic engineered nucleases are packaged into a delivery vehicle (virus, liposomes, naked-DNA, etc) and injected directly into the patient