Gene-circuit therapy on the horizon: synthetic biology tools for engineered therapeutics

Rafał Krzysztoń; Yiming Wan; Julia Petreczky; Gábor  Balázsi

doi:10.18388/abp.2020_5744

Rafał Krzysztoń 1Biomedical Engineering Department, Stony Brook University, Stony Brook, NY 11974, USA; 2The Louis and Beatrice Laufer Center for Physical and Quantitative Biology, Stony Brook University, Stony Brook, NY 11794, USA
Yiming Wan 1Biomedical Engineering Department, Stony Brook University, Stony Brook, NY 11974, USA; 2The Louis and Beatrice Laufer Center for Physical and Quantitative Biology, Stony Brook University, Stony Brook, NY 11794, USA
Julia Petreczky 1Biomedical Engineering Department, Stony Brook University, Stony Brook, NY 11974, USA; 2The Louis and Beatrice Laufer Center for Physical and Quantitative Biology, Stony Brook University, Stony Brook, NY 11794, USA
Gábor Balázsi 1Biomedical Engineering Department, Stony Brook University, Stony Brook, NY 11974, USA; 2The Louis and Beatrice Laufer Center for Physical and Quantitative Biology, Stony Brook University, Stony Brook, NY 11794, USA

DOI: https://doi.org/10.18388/abp.2020_5744

Abstract

Therapeutic genome modification requires precise control over the introduced therapeutic functions. Current approaches of gene and cell therapy fail to deliver such command and rely on semi-quantitative methods with limited influence on timing, contextuality and levels of transgene expression, and hence on therapeutic function. Synthetic biology offers new opportunities for quantitative functionality in designing therapeutic systems and their components. Here, we discuss synthetic biology tools in their therapeutic context, with examples of proof-of-principle and clinical applications of engineered synthetic biomolecules and higher-order functional systems, i.e. gene circuits. We also present the prospects of future development towards advanced gene-circuit therapy.