Advances in HIV Gene Therapy

Abstract

Early gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy product targeting genetic mutations in 2017. Gene therapy (GT) can be delivered either in vivo or ex vivo. An ex vivo approach to gene therapy is advantageous, as it allows for the characterization of the gene-modified cells and the selection of desired properties before patient administration. Autologous cells can also be used during this process which eliminates the possibility of immune rejection. This review highlights the various stages of ex vivo gene therapy, current research developments that have increased the efficiency and safety of this process, and a comprehensive summary of Human Immunodeficiency Virus (HIV) gene therapy studies, the majority of which have employed the ex vivo approach.

CRISPR technology in human diseases

“… 376 HIV infection of cells of the host immune system integrates its genetic material onto human cells for a short period, forming a stable, latent state viral reservoir, ART is difficult to absorb by tissues carrying the latent virus, and the virus may rebound weeks after cessation of treatment. 377 Elimination of viral reservoirs with integrated replication capacity in host cell DNA using CRISPR–Cas9 gene editing is a promising strategy for achieving a functional cure for HIV. 378 , 379 The complete elimination of HIV‐1 has become a major point of research to keep the virus from recurring.…”