Module 2: In vivo gene therapy

Lecture 7: In-situ, in-vivo and ex-vivo gene therapy (part I)

    

Figure 7.3 Clinical ex vivo and in vivo approach:

In vivo, the transgene can be directly injected by various routes based on the clinical condition of the patient and design of the vector. For instance, transducing the lung airway epithelium is targeted. The target specific gene therapy is possible with the help of unique promoters. The liver directed gene therapy is done usually by thyroid binding globulin promoter (TBG) while cytomegalovirus (CMV) promoter can be used to direct the muscle cells. Ex vivo on the other hand requires the manipulation of the cells outside the body and the transducer cells are injected back after quality assurance.