Small RNA molecules such as microRNA and small interfering RNA (siRNA) have become promising therapeutic agents because of their specificity and their potential to modulate gene expression. Any gene of interest can be potentially up- or down-regulated, making RNA-based technology the healthcare breakthrough of our era. However, the functional and specific delivery of siRNAs into tissues of interest and into the cytosol of target cells remains highly challenging, mainly due to the lack of efficient and selective delivery systems. Among the variety of carriers for siRNA delivery, peptides have become essential candidates because of their high selectivity, stability, and conjugation versatility. Here, we describe the development of molecules encompassing siRNAs against SOD1, conjugated to peptides that target the low-density lipoprotein receptor (LDLR), and their biological evaluation both in vitro and in vivo.

中文翻译：

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LDLR 介导的体外和体内 siRNA-肽配体缀合物的靶向和生产性摄取

小RNA分子，如microRNA和小干扰RNA (siRNA)，因其特异性和调节基因表达的潜力而成为有前途的治疗剂。任何感兴趣的基因都可能被上调或下调，这使得基于 RNA 的技术成为我们这个时代的医疗保健突破。然而，将 siRNA 功能性且特异性地递送到感兴趣的组织和靶细胞的细胞质中仍然具有高度挑战性，这主要是由于缺乏有效和选择性的递送系统。在用于 siRNA 递送的各种载体中，肽因其高选择性、稳定性和缀合多功能性而成为重要的候选者。在这里，我们描述了包含针对 SOD1 的 siRNA 的分子的开发，这些分子与靶向低密度脂蛋白受体 (LDLR) 的肽缀合，以及它们的体外和体内生物学评估。