AReversion of chromosomal inversionEndothelin cellsRestoration of factor VIII expression, rescue of lethality in mouse xenograft model(Park et al. 2015) Severe combined immunodeficiency (SCID)Repair of loss of function mutation (JAK3)T-cellsRestoration of correct T-cell development(Chang et al. 2015) Sickle Cell AnaemiaReversion of point mutation (HBB)ErythrocytesHBB expression(Huang et al. 2015)
