Cerrato Carmine Pasquale, Künnapuu Kadri, Langel Ülo
a Department of Neurochemistry , Stockholm University , Stockholm , Sweden.
b Laboratory of Molecular Biotechnology, Institute of Technology , University of Tartu , Tartu , Estonia.
Expert Opin Drug Deliv. 2017 Feb;14(2):245-255. doi: 10.1080/17425247.2016.1213237. Epub 2016 Aug 2.
One of the major limiting steps in order to have an effective drug is the passage through one or more cell membranes to reach its site of action. To reach the action-site, the specific macromolecules are required to be delivered specifically to the cell compartment/organelle in their (pre)active form. Areas covered: In this review, we will discuss cell-penetrating peptides (CPPs) developed in the last decade to transport small RNA/DNA, plasmids, antibodies, and nanoparticles into specific sites of the cell. The article describes CPPs in complex with cargo molecules that target specific intracellular organelles and their potential for pharmacological or clinical use. Expert opinion: Organelle targeting is the ultimate goal to ensure selective delivery to the site of action in the cells. CPP technologies represent an important strategy to address drug delivery to specific intracellular compartments by covalent conjugation to targeting sequences, potentially enabling strategies to combat genomic diseases as well as infections, cancer, neurodegenerative and hereditary diseases. They have proven to be successful in delivering various therapeutic agents into cells however, further in vivo experiments and clinical trials are required to demonstrate the efficacy of this technology.
获得一种有效药物的主要限制步骤之一是穿过一层或多层细胞膜以到达其作用位点。为了到达作用位点，特定的大分子需要以其（预）活性形式被特异性递送至细胞区室/细胞器。涵盖领域：在本综述中，我们将讨论过去十年中开发的细胞穿透肽（CPP），其用于将小RNA/DNA、质粒、抗体和纳米颗粒转运至细胞的特定部位。本文描述了与靶向特定细胞内细胞器的货物分子复合的CPP及其药理学或临床应用潜力。专家观点：细胞器靶向是确保选择性递送至细胞内作用位点的最终目标。CPP技术是通过与靶向序列共价缀合来解决向特定细胞内区室进行药物递送的重要策略，有可能实现对抗基因组疾病以及感染、癌症、神经退行性疾病和遗传性疾病的策略。它们已被证明在将各种治疗剂递送至细胞方面是成功的，然而，需要进一步的体内实验和临床试验来证明该技术的有效性。
