Laboratory for Research on Neurodegenerative Disorders, IRCCS Maugeri Clinical and Scientific Institutes SpA SB, Via Maugeri 10, 27100 Pavia, Italy.
Laboratory for Research on Neurodegenerative Disorders, IRCCS Maugeri Clinical and Scientific Institutes SpA SB, Via Maugeri 10, 27100 Pavia, Italy.
Trends Pharmacol Sci. 2017 Apr;38(4):406-424. doi: 10.1016/j.tips.2017.01.003. Epub 2017 Feb 14.
The presence of cell and tissue barriers together with the low biomembrane permeability of various therapeutics often hampers systemic drug distribution; thus, most of the available molecules are of limited therapeutic value. Opportunities to increase medicament concentrations in areas that are difficult to access now exist with the advent of cell-penetrating peptides (CPPs), which can transport into the cell a wide variety of biologically active conjugates (cargoes). Numerous preclinical evaluations with CPP-derived therapeutics have provided promising results in various disease models that, in some cases, prompted clinical trials. The outcome of these investigations has thus opened new perspectives for CPP application in the development of unprecedented human therapies that are well tolerated and directed to intracellular targets.
细胞和组织屏障的存在以及各种治疗药物的生物膜通透性低，常常阻碍全身药物分布；因此，大多数现有的分子的治疗价值有限。随着穿透细胞肽（CPPs）的出现，现在有机会增加难以到达的区域的药物浓度，这些肽可以将各种生物活性缀合物（货物）输送到细胞内。大量使用 CPP 衍生的治疗剂进行的临床前评估在各种疾病模型中取得了有希望的结果，在某些情况下，这些结果促使进行了临床试验。这些研究的结果为 CPP 在开发前所未有的人类疗法中的应用开辟了新的前景，这些疗法具有良好的耐受性，并针对细胞内靶标。
