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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]
In science, ex vivo refers to experimentation or measurements done in or on tissue from an organism in an external environment with minimal alteration of natural conditions. [ 2 ] A primary advantage of using ex vivo tissues is the ability to perform tests or measurements that would otherwise not be possible or ethical in living subjects.
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.
Gene therapy seeks to modulate or otherwise affect gene expression via the introduction of a therapeutic transgene. Gene therapy by viral vectors can be performed by in vivo delivery by directly administering the vector to the patient, or ex vivo by extracting cells from the patient, transducing them, and then reintroducing the modified cells ...
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.
Strimvelis is the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA). [2] Medical uses.
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.
How vectors work to transfer genetic material. Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).