Department of Pharmaceutical Sciences, University of Connecticut, Storrs, CT, United States.
Biomedical Engineering Department, University of Connecticut, Storrs, CT, United States.
Curr Pharm Des. 2018;24(43):5164-5174. doi: 10.2174/1381612825666190117164901.
Peptide nucleic acids (PNAs) belong to the next generation of synthetic nucleic acid analogues. Their high binding affinity and specificity towards the target DNA or RNA make them the reagent of choice for gene therapy-based applications.
To review important gene therapy based applications of regular and chemically modified peptide nucleic acids in combination with nanotechnology.
Selective research of the literature.
Poor intracellular delivery of PNAs has been a significant challenge. Among several delivery strategies explored till date, nanotechnology-based strategies hold immense potential. Recent studies have shown that advances in nanotechnology can be used to broaden the range of therapeutic applications of PNAs. In this review, we discussed significant advances made in nanoparticle-based on PLGA polymer, silicon, oxidized carbon and graphene oxide for the delivery of PNAs.
Nanoparticles delivered PNAs can be implied in diverse gene therapy based applications including gene editing as well as gene targeting (antisense) based strategies.
肽核酸(PNA)属于下一代合成核酸类似物。它们与目标 DNA 或 RNA 的高结合亲和力和特异性使其成为基因治疗应用的首选试剂。
综述常规和化学修饰的肽核酸与纳米技术结合在基因治疗中的重要应用。
选择性文献研究。
PNA 的细胞内递送效果不佳一直是一个重大挑战。在迄今为止探索的几种递送策略中,基于纳米技术的策略具有巨大的潜力。最近的研究表明,纳米技术的进步可用于拓宽 PNA 的治疗应用范围。在本综述中,我们讨论了基于 PLGA 聚合物、硅、氧化碳和氧化石墨烯的纳米粒子在递送 PNA 方面的重要进展。
递送到细胞内的 PNA 纳米粒可应用于多种基因治疗,包括基因编辑以及基因靶向(反义)策略。