HIV Gene Therapy: An Update

Hum Gene Ther. 2021 Jan;32(1-2):52-65. doi: 10.1089/hum.2020.159.

Abstract

Progress in antiretroviral therapy has considerably reduced mortality and notably improved the quality of life of individuals infected with HIV since the pandemic began some 40 years ago. However, drug resistance, treatment-associated toxicity, adherence to medication, and the need for lifelong therapy have remained major challenges. While the development of an HIV vaccine has remained elusive, considerable progress in developing innovative cell and gene therapies to treat HIV infection has been made. This includes immune cell therapies, such as chimeric antigen receptor T cells to target HIV infected cells, as well as gene therapies and genome editing strategies to render the patient's immune system resistant to HIV. Nonetheless, all of these attempts to achieve a functional cure in HIV patients have failed thus far. This review introduces the clinical as well as the technical challenges of treating HIV infection, and summarizes the most promising cell and gene therapy concepts that have aspired to bring about functional cure for people living with HIV. It further discusses socioeconomic aspects as well as future directions for developing cell and gene therapies with a potential to be an effective one-time treatment with minimal toxicity.

Keywords: AIDS; CCR5; CRISPR-Cas; epigenome editing; gene editing; latent HIV reservoir.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Gene Editing
  • Genetic Therapy
  • HIV Infections* / genetics
  • HIV Infections* / therapy
  • Humans
  • Quality of Life