CMV-iCre-Puro Lentivirus

Lentiviral vectors (LVs) are replication-defective mixed-enveloped viral particles composed of a minimal set of capsid proteins from a parental virus (most often HIV), surface proteins from an unrelated virus (called a pseudotype), and a recombinant viral genome containing cis-acting elements of the parental virus necessary for gene transfer, as well as a transgene expression cassette of choice. The vesicular stomatitis virus surface glycoprotein (VSV.G) is commonly used to pseudotype LVs because of its high stability and broad tropism. LVs are emerging as powerful and versatile gene delivery vectors due to i) their ability to efficiently transfer (i.e., transduce) genes into a variety of dividing and non-dividing cell types and to stably integrate their genome into target cell chromatin; ii) their relatively large cargo capacity; and iii) low human immunity to vector components compared to other virus-derived gene transfer vectors.

Currently, LVs are involved in 7% of gene therapy clinical trials worldwide, as well as 19% of clinical trials for monogenic diseases. LVs have been used for two gene therapy applications: ex vivo gene therapy (where target cells are harvested from the recipient, genetically modified, and then infused back into the recipient) and in vivo gene therapy (where LVs are injected directly into the recipient, either locally, such as the brain or eyes, or systemically to reach organs such as the liver or spleen). Ex vivo LV gene therapy using hematopoietic stem and progenitor cells (HSPCs) or T cells has entered late-stage clinical trials, whereas in vivo LV gene therapy is mostly in preclinical development.