人性化小鼠HIV-1基因治疗的教训：以病毒为靶点可行吗？

Title:Lessons From HIV-1 Gene Therapy in Humanized Mice: Is Targeting Viral Entry the Road to Success?

Volume: 16 Issue: 1

Author(s): Nicolas Petit and Gilles Marodon

Affiliation:

关键词: HIV-1

摘要: Immunodeficient mice reconstituted with human CD4⁺ T cells, which can be achieved either by transfer of mature cells or immature progenitors, represent the only animal model to study HIV-1 infection of human lymphocytes in vivo. However, the immunocompromised status of most of these models currently rule out their use for vaccine studies. Nevertheless, the model might be ideally suited for HIV-1 gene therapy studies since eliciting an efficient anti-viral immune response is not the primary end-point. Rather, HIV-1 gene therapy should protect CD4⁺ T cells from HIV-1- induced deletion and/or reduced viral replication. Here, we describe recent advancements in the field of HIV-1 gene therapy, focusing on tools and targets validated in various models of humanized mice. From the analysis of this literature, it appears that strategies targeting viral entry, by means of neutralizing antibodies or fusion inhibitors, are the most promising so far. Indeed, strategies targeting viral entry have moved to the clinic with encouraging results. Thus, humanized mice should be considered as the prime model to devise the safer and most effective HIV-1 gene therapy strategy.

Cite this article as:

Nicolas Petit and Gilles Marodon , Lessons From HIV-1 Gene Therapy in Humanized Mice: Is Targeting Viral Entry the Road to Success?, Current Gene Therapy 2016; 16 (1) . https://dx.doi.org/10.2174/1566523216666160104141644