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In ex vivo gene therapies, such as CAR-T therapeutics, the patient's own cells (autologous) or healthy donor cells (allogeneic) are modified outside the body (hence, ex vivo) using a vector to express a particular protein, such as a chimeric antigen receptor. [62]
Strimvelis is the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA). [2] Medical uses.
Experiments generally start after 24 hours of incubation. Using living cells or tissue from the same organism are still considered to be ex vivo. One widely performed ex vivo study is the chick chorioallantoic membrane (CAM) assay. In this assay, angiogenesis is promoted on the CAM membrane of a chicken embryo outside the organism (chicken).
Two methods of gene therapy using lentiviruses have been proposed. In the ex vivo methodology, cells are extracted from a patient and then cultured. A lentiviral vector carrying therapeutic transgenes are then introduced to the culture to infect them. The now modified cells continue to be cultured until they can be infused into the patient.
The majority of gene therapy approaches leverage viral vectors, such as adeno-associated viruses (AAVs), adenoviruses (AV), and lentiviruses (LV), to facilitate the insertion or replacement of transgenes either in vivo or ex vivo. These vectors serve as delivery vehicles for introducing the therapeutic genetic material into the patient's cells.
Central line insertion of catheter. This is the most common method of infusion of CAR-T cells to systemic circulation after ex-vivo manufacturing. The classic method of administration of CAR-T cells to cancers within the human body is through intravenous (IV) central line infusion. [6]
In diseases that are secondary to a genetic mutation that causes the lack of a gene, the gene is added back in. [24] [25] [26] In diseases that are due to the overexpression of a gene, viral genetic engineering may be introduced to turn off the gene. [24] [25] [26] Viral gene therapy may be done in vivo or ex vivo.
The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include repairing an abnormal gene and altering the degree to which a gene is turned on or off. Two basic methodologies are utilized to transfer vectors into target tissues; Ex vivo gene transfer and In vivo gene transfer.