Viral Vectors in Gene Therapy

In gene therapy, viral and non-viral vectors are used to deliver a therapeutic gene to a patient's cells.

Viruses are the perfect tool for nucleic acid delivery as they naturally evolved to bring in their genetic material into cells to replicate. Some viruses, such as retroviruses, even insert their genes into the host cells genome. In order to ensure the safety of the viral vectors, either the whole viral genome is replaced by the therapeutic gene or the parts that are disease-causing are deleted from the viral vector.

Different types of viral vectors exist:

Figure 1: Incorporation of a therapeutic gene into the viral vector adeno-associated virus (AAV).