Department of Chemistry, University of Rochester, Rochester, NY, USA.
Department of Pediatrics and Neonatology, Biomedical Engineering, and Pharmacology & Physiology, School of Medicine and Dentistry, University of Rochester, Rochester, NY, USA.
Methods Mol Biol. 2021;2208:49-67. doi: 10.1007/978-1-0716-0928-6_4.
The potential of RNAi therapies has been largely impeded by the inherent challenges in the functional delivery of siRNA to cells. Herein, we describe protocols for the synthesis and characterization of novel peptide-siRNA nanoparticles prepared from disulfide-constrained amphipathic peptides complexed with siRNA as promising siRNA delivery vectors. We also describe protocols for the application of these nanoparticles to the in vitro and in vivo delivery of siRNA to lung cells for the functional knockdown of lung proteins.
RNAi 疗法的潜力在很大程度上受到将 siRNA 递送到细胞中的固有挑战的阻碍。在此，我们描述了从二硫键约束的两亲肽合成和表征新型肽-siRNA 纳米颗粒的方案，这些肽-siRNA 纳米颗粒与 siRNA 复合，作为有前途的 siRNA 递药载体。我们还描述了将这些纳米颗粒应用于体外和体内递送至肺细胞中的 siRNA，以实现肺蛋白的功能敲低的方案。
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