EF1a-FLPo-Puro Lentivirus

After 20 years of clinical trials, gene therapy has shown effectiveness in treating a range of diseases. Currently, there are several gene therapy products approved and in clinical use. The most commonly used virus in gene therapy is derived from human immunodeficiency virus type 1 (HIV-1), although several non-primate alternative viruses have been developed, such as feline immunodeficiency virus (FIV) or equine infectious anemia virus (EIAV). The ability of lentiviruses to transduce both proliferating and non-proliferating cells and the long-term expression they provide through genomic integration make them the vector of choice in several gene therapies.

Lentiviral vectors (LVs) are one of the most commonly used vectors for delivering genetic material and are the vector of choice for modifying hematopoietic cells to correct primary immunodeficiencies, hemoglobinopathies, and leukodystrophies. In adoptive T-cell cancer therapy, autologous T lymphocytes are genetically modified to express T cell receptors (TCRs) or chimeric antigen receptors (CARs) to expand or redirect their antigenic repertoire to malignant cells. LVs are also widely used to modify T cells to treat cancer in immunotherapy (e.g., chimeric antigen receptor T-cell therapy, CAR-T). In the past 5 years, two CAR-T LV-based therapeutic products have received market approval. In genome editing, LVs are used to deliver sequence-specific designer nucleases and DNA templates. Approval of LV gene therapy products (such as Kymriah, for the treatment of B-cell acute lymphoblastic leukemia.