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From edits to Circuits: Nucleic acid therapeutics for Correcting human disease
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DOI：10.1016/j.mattod.2026.103351.png)
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Abstract
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Nucleic acid therapeutics (NATs) allow replacement, modulation, and elimination of entire genes or their products. These molecules are diverse in type, materializing as RNA, DNA, or other forms, and can induce either temporary or permanent effects at the cellular and organ level. Furthermore, these therapies can be administered directly in vivo or applied ex vivo to tissues or cells that are subsequently reintroduced into patients. To achieve therapeutic distribution, delivery vehicles carrying these payloads are just as diverse for transporting NATs to specific cells, tissues, and organs. Here, we focus on three main areas of in vivo NATs: 1) what types of NATs exist, 2) what are the delivery vehicles used to protect or carry them, and 3) what is the clinical landscape these tools operate within. Additionally, this review highlights how NATs are increasingly defined by organ-level targeting. The evolution of NATs for gene editing to knockout, silencing, or upregulation are also discussed for single-gene modulation to programmable gene circuits, whose safety and efficacy depend on precise delivery and controlled persistence. As delivery technologies extend beyond liver-dominated biodistribution and enable tighter spatial and temporal control, nucleic acid therapeutics are positioned to evolve from corrective interventions into responsive, programmable medicines. We conclude with upcoming milestones and future directions for these technologies.
Keywords:
Gene therapy
Nucleic acid therapy
Cell therapy
Synthetic biology
Genetic circuits
Delivery vehicles
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