Fig. 2
From: Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches
Ex-vivo GT approach for MPS. In ex-vivo GT approaches, patient cells are collected and stem cells are isolated; thereafter, they are mixed with the viral vector in which the therapeutic gene has been inserted. The final transduced stem cells are later re-infused in the patient after administration of a conditioning regimen and, by engrafting in the patient tissues, are able to restore the healthy phenotype. Modified from Penati et al., J Inherit Metab Dis. 2017, in press