Towards Self-Transfecting Nucleic Acid Nanostructures for Gene Regulation

摘要

Nanoscale structures of therapeutic nucleic acids have shown enormous potential to help clinicians realize the promise of personaliz ed medicine using gene-specific treatments. With the advent of better sequencing through bioinformatic approaches and advancements in nucleic acid stabilization chemistries, the field of synthetic nucleic acid nanomaterials has advanced tremendously. This review focuses on an emerging strategy geared at gene silencing without the use of traditional polycation-based transfection agents and discusses how such nanostructures are being chemically tailored to navigate biological systems to improve their circulation time and biodistribution. We also address important challenges moving forward, including quantification of delivery and the multiplexing of sequences for regulating gene networks - a goal well suited for this unique class of materials.