A common vehicle used extensively in gene therapy is the lentiviral vector (LV). The LV is a replication-defective viral vector, generated from HEK293T cells (human embryonic kidney cells) after the transfection with plasmids (DNA molecules) dedicated for the assembling of particular viral parts (envelope, capsid, nucleoproteins, viral RNA, reverse transcriptase, integrase etc). The purified particles can be used for ex vivo gene delivery, after the inoculation of cells of interest harvested from the patient/donor. Upon entry, the two viral single-stranded RNA molecules are reverse transcribed and integrated into the cellular genome. The transduced cells can be expanded or directly infused back to the recipient patient. Ideally, the expression of a therapeutic gene will result in significantly reduced disease manifestations or even cure of the patients.
Author: Filippos T Charitidis (ESR9)