摘要
Given its ability to knock down essentially any gene of interest, siRNA has been one of the promising candidates for gene therapy. However, like other nucleic-acid-based drugs, its poor cellular uptake poses a major challenge. Here we briefly summarize the use of cell penetrating peptides (CPPs) as a novel and promising approach for siRNA delivery. The main advantages of CPPs are their low toxicity and high efficiency.
siRNA被认为是很有希望的基因治疗分子,它通过介导靶mRNA降解而抑制基因表达。为了发挥siRNA分子的应用,需要寻找适当的方法解决siRNA分子透膜性差的问题。用细胞透膜性肽进行药物细胞内转运是一种新的和充满希望的方法。本文将综述细胞透膜性肽在siRNA转运中的应用。初步研究表明这类转运载体具有较低的细胞毒性和较好的转运效率。