Gene therapy vector

A gene therapy vector is a specialized delivery vehicle designed to transport genetic material, such as DNA or RNA, into a patient's cells to achieve a therapeutic effect. These vectors are broadly categorized into viral types, including adeno-associated virus (AAV), lentivirus, and adenovirus, and non-viral types, such as lipid nanoparticles (LNPs) and polymer-based systems (NIH, 2023). Their primary biological role is to overcome cellular barriers to deliver genetic payloads that can replace defective genes, knock down overexpressed genes, or introduce novel functions like chimeric antigen receptors in T cells (FDA, 2022). In clinical practice, they are utilized to treat a variety of conditions, including spinal muscular atrophy, inherited retinal diseases, and various malignancies. The interaction with the host involves complex processes like endocytosis, endosomal escape, and nuclear entry. However, safety concerns such as pre-existing immunity to viral capsids, potential insertional mutagenesis, and systemic inflammatory responses remain significant hurdles in the field (Nature Reviews Drug Discovery, 2019). Monitoring efficacy and safety often requires assessing neutralizing antibody titers and vector copy numbers in the patient.

Other names

Gene delivery vehicleViral vectorNon-viral vectorGenetic carrierGene delivery system

Delivery of genetic material to host cells via viral transduction or non-viral transfection to enable therapeutic protein expression or gene silencing (NIH, 2023; FDA, 2022).