Vector

The "vehicle" or carrier for delivering genes or genetic information into the cell, particularly useful for gene therapy.

A vector is the vehicle for delivering genes or genetic information into the nucleus of a cell. Vectors act like a fleet of microscopic delivery trucks transporting new, therapeutic ("good") genes into retinal cells. Most vectors are modified forms of viruses (a "safe" form). Viruses are extremely effective at getting inside a cell; however, viruses also contain elements that may damage cells. Over the past two decades, researchers have been working to genetically modify viruses to be safe and have few immune side effects, without compromising their delivery capabilities. Examples of viral vectors include: lentiviral vectors, adeno-associated vectors (AAV), and encapsidated adenovirus minichromosome (EAM). The clinical trials for LCA (RPE65) gene therapy use retina-specific AAV vectors.