Targeted Gene Therapy Via Lentiviral Vectors

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Introduction

By Drew Albrecht
Gene therapy is a medicinal technique that is on the forefront of medical innovation. With the use of targeted gene therapy, disease-specific treatments can be made to cure people of diseases that were once thought to be incurable. There are many methods that are being tested right now to advance gene therapy, but one that is gaining serious traction are lentiviral vectors. Vectors based on viruses are very attractive for gene therapy because viruses are naturally very good at getting into cells. Lentiviruses are even better because they insert their genes into the host’s genomes. With the creation of lentiviral particles with a specific gene of interest, researchers can target affected cells and recover the corrected DNA. This methodology is already being used to treat and cure many diseases today, and the future holds many promises for beating the unbeatable.

Gene Therapy Overview

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

Viral Vectors

Lentiviruses

Lentivirus Infection Pathway

Figure ?. Infection Pathway of HIV-1 Lentivirus. Perilla et al. (2022)

Lentiviral Vectors

Figure ?. Simplified schematic of the HIV-1 structure and respective proteins. Rittiner et al. (2020)

How Do Lentivirus Vectors Work

Figure ?. The core plasmids and transfer plasmid that are packaged into a lentiviral particle. Milone and O'Doherty (2018)

Figure ?. Functions of known lentivirus proteins. Genemedi

HIV As a Viral Vector

Using HIV To Fight HIV

Figure ?. Timothy Ray Brown, one of the only cured survivors of HIV/AIDS. NIH

Current Treatments

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