Peer Dan
Laboratory of NanoMedicine, Department of Cell Research and Immunology, George S. Wise Faculty of Life Science, Tel Aviv, 69978 Israel ; Department of Materials Science and Engineering, Faculty of Engineering, Tel Aviv, 69978 Israel ; Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, 69978 Israel.
Mol Cell Ther. 2014 Feb 5;2:5. doi: 10.1186/2052-8426-2-5. eCollection 2014.
Utilizing RNA interference as an innovative therapeutic strategy has an immense likelihood to generate novel concepts in precision medicine. Several clinical trials are on the way with some positive initial results. Yet, targeting of RNAi payloads such as small interfering RNAs (siRNAs), microRNA (miR) mimetic or anti-miR (antagomirs) into specific cell types remains a challenge. Major attempts are done for developing nano-sized carriers that could overcome systemic, local and cellular barriers. This progress report will focus on the recent advances in the RNAi world, detailing strategies of systemic passive tissue targeting and active cellular targeting, which is often considered as the holy grail of drug delivery.
将RNA干扰作为一种创新的治疗策略极有可能在精准医学中产生新的理念。几项临床试验正在进行中，并取得了一些积极的初步成果。然而，将RNA干扰有效载荷（如小干扰RNA（siRNAs）、微小RNA（miR）模拟物或抗miR（拮抗剂））靶向特定细胞类型仍然是一个挑战。目前正在进行大量尝试，以开发能够克服全身、局部和细胞屏障的纳米级载体。本进展报告将聚焦于RNA干扰领域的最新进展，详细阐述全身被动组织靶向和主动细胞靶向策略，而主动细胞靶向通常被视为药物递送的圣杯。
