Hematopoietic-stem-cell-based gene therapy for HIV disease

Cell Stem Cell. 2012 Feb 3;10(2):137-47. doi: 10.1016/j.stem.2011.12.015.

Abstract

Although combination antiretroviral therapy can dramatically reduce the circulating viral load in those infected with HIV, replication-competent virus persists. To eliminate the need for indefinite treatment, there is growing interest in creating a functional HIV-resistant immune system through the use of gene-modified hematopoietic stem cells (HSCs). Proof of concept for this approach has been provided in the instance of an HIV-infected adult transplanted with allogeneic stem cells from a donor lacking the HIV coreceptor, CCR5. Here, we review this and other strategies for HSC-based gene therapy for HIV disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adult
  • Animals
  • Gene Knockout Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors
  • HIV / genetics
  • HIV / physiology*
  • HIV Infections / genetics
  • HIV Infections / immunology
  • HIV Infections / pathology
  • HIV Infections / therapy*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immune System
  • Receptors, CCR5 / genetics

Substances

  • Receptors, CCR5