Gene Therapy Strategies Targeting Aging-Related Diseases

Abstract

Rapid advancements have taken place in gene therapy technology. However, effective methods for treating aging- or age-related chronic diseases, which are often closely related to genes or even multiple genes, are still lacking. The path to developing cures is winding, while gene therapy that targets genes related to aging represents an exciting research direction with tremendous potential. Among aging-related genes, some candidates have been studied at different levels, from cell to organismal levels (e.g., mammalian models) with different methods, from overexpression to gene editing. The TERT and APOE have even entered the stage of clinical trials. Even those displaying only a preliminary association with diseases have potential applications. This article discusses the foundations and recent breakthroughs in the field of gene therapy, providing a summary of current mainstream strategies and gene therapy products with clinical and preclinical applications. Finally, we review representative target genes and their potential for treating aging or age-related diseases.

Keywords: CRISPR; aging; aging-related disease; gene replacement; gene therapy.

Figure 1.

Strategies of gene therapy. The schematic enumerates the strategies of gene therapy summarized in this review.

Figure 2.

Schematic of the cellular components of proteins encoded by representative aging-related genes.