Development of siRNA Nano-Carriers for In Vivo R&D and Gene Therapy

用于体内 R 的 siRNA 纳米载体的开发

• 批准号: 710195
• 金额: $ 7.64万
• 依托单位: DELIVERICS LIMITED
• 依托单位国家: 英国
• 项目类别: GRD Proof of Concept
• 财政年份: 2012
• 资助国家: 英国
• 起止时间: 2012 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=710195
• 关键词: Development; siRNA; Nano; Carriers; Vivo

Nucleic acids are at the very center of all cellular processes, with chromosomal DNA serving as a storage device for genetic information and RNA molecules directly controlling gene expression and, through this, each and every cellular function. Consequently, the clinical application of short RNA molecules (e.g. miRNA, siRNA) to “silence” the expression of specific malignant genes (process known as RNA interference) can be used to treat both genetic and acquired diseases (e.g. cancers and viral infections) at their origin, not merely their symptoms. Stimulated by its therapeutic potential and outstanding advances in preclinical gene therapy and regenerative medicine, the use of RNA interference (RNAi) in R&D and Pharma is a fast growing sector of the biomedical market. There is, however, a major technical hurdle: the transport of nucleic acids through cell membranes is blocked by their size and negative charge. To facilitate the intracellular delivery of siRNA, DELIVERICS developed a ground-breaking technology based on biodegradable nano-carriers able to carryout this challenging task at very high efficiency without toxicity; a technology currently commercialised as R&D kits for “in vitro” studies: SAFEctin Transfection Reagent and SAFEctin-STEM. Aiming to develop a gene therapy drug able to treat skin lesions by topical administration, DELIVERICS (in collaboration with the University of Edinburgh) aims to optimise its platform technology for siRNA delivery into skin cells. This biodegradable vehicle, so-called LIFEctin LOCAL, will address a commercial opportunity that exists in both the R&D and Pharma sectors, where siRNA delivery tools with clinical-grade safety and reliability are required for "in vivo" R&D studies and for developing efficient RNAi-based drugs.

核酸是所有细胞过程的核心，染色体DNA作为遗传信息的存储设备，RNA分子直接控制基因表达，并通过它控制每一个细胞功能。因此，临床应用短RNA分子（如miRNA、siRNA）“沉默”特定恶性基因的表达（称为RNA干扰的过程）可用于从源头治疗遗传和获得性疾病（如癌症和病毒感染），而不仅仅是治疗其症状。受其治疗潜力和临床前基因治疗和再生医学的杰出进展的刺激，RNA干扰（RNAi）在研发和制药中的应用是生物医学市场中快速增长的领域。然而，有一个主要的技术障碍：核酸通过细胞膜的运输受到它们的大小和负电荷的阻碍。为了促进siRNA的细胞内递送，DELIVERICS开发了一种基于可生物降解纳米载体的突破性技术，能够以非常高的效率而无毒地完成这项具有挑战性的任务；目前商业化的研发试剂盒用于“体外”研究：SAFEctin转染试剂和SAFEctin- stem。DELIVERICS（与爱丁堡大学合作）旨在开发一种能够通过局部给药治疗皮肤病变的基因治疗药物，旨在优化其将siRNA递送到皮肤细胞的平台技术。这种可生物降解的载体，称为LIFEctin LOCAL，将解决研发和制药领域存在的商业机会，在“体内”研发研究和开发有效的基于rna的药物中，需要具有临床安全性和可靠性的siRNA递送工具。