Targeted Gene Therapy Via Lentiviral Vectors

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Gene Therapy Overview

Figure 1. Schematic drawing of ex vivo and in vivo gene therapy Bulcha et al. (2021)

By Drew Albrecht

Genetic disorders like cystic fibrosis, phenylketonuria, sickle-cell anemia, and many more, are a result of mutated or absent genes. The goal of gene therapy is to treat or cure such diseases via genetic modification of the cells in an affected individual. Genetic modification can rewire the cells to either produce a therapeutic effect[Kaji] or simply just replace the mutated or missing DNA[Lai]. Gene therapy can either be ex vivo, where genetic modification of cells occurs outside of the body followed by their transplantation back into the body[Gowing] or in vivo, when genetic information is directly inserted into the body via a vector[Mendell]. Vectors are molecules that aid in the transportation of genetic information all over the body. While both methods prove useful for fighting diseases with genetic modification, in vivo is the more appropriate method for a targeted approach. Performing in vivo gene therapy, there are two different types of vectors: nonviral and viral. Non-viral vectors can be naked DNA, particle based, or chemical based, all transporting DNA throughout the body without the use of a virus. Like the name suggests, viral vectors use the natural tendencies of viruses to infect a host by inserting its DNA into the cytoplasm. Viruses are the perfect carrier to deliver DNA to a target because that is what they do naturally. While non-viral are overall less effective, they are still a promising option due to the low cost, reduced pathogenicity, and ease of production. However, there are a few types of viral vectors that are being tested to drastically change the face of medicine in disease treatment. Currently, the three types of viruses that are being used are adenoviruses (AVs), and adeno-associated viruses (AAVs), and retroviruses, also called lentiviruses.

Viral Vectors

Lentiviruses

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