Fig. 2: Schematic of ex vivo and in vivo strategies (shown in blue boxes) for treating genetic diseases. | Nature Communications

Fig. 2: Schematic of ex vivo and in vivo strategies (shown in blue boxes) for treating genetic diseases.

From: The once and future gene therapy

Fig. 2

For ex vivo approaches (left panel), autologous cells can be isolated directly from the patient and genetically modified to elicit a therapeutic effect, while allogeneic cells can be produced and readily available “off the shelf.” In vivo strategies require targeting of specific cells in order to overexpress a therapeutic gene or correct pathological mechanisms to allow functional gene expression (dashed arrows).

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