SBIR Phase II: Improved in Vivo Delivery of SiRNA

SBIR II 期：SiRNA 体内递送的改进

• 批准号: 0923854
• 负责人: Lance Ford
• 金额: $ 50万
• 依托单位: Bioo Scientific Corporation
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-08-01 至 2013-06-30
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=0923854&HistoricalAwards=false
• 关键词: SBIR; Phase; II; Improved; Vivo

This award is funded under the American Recovery and Reinvestment Act of 2009 (Public Law 111-5).This Small Business Innovation Research (SBIR) Phase II project will develop technologies that optimize the use of RNA interference (RNAi) in animals. RNAi is an invaluable tool for characterizing gene function and is a promising candidate for gene therapy. The use of RNAi in tissue culture is well developed but is of limited use in experimental animals. RNAi agents must enter cells to exert their effects but this has proven to be challenging in animals. The current lack of such technologies is holding back the majority of important RNAi animal experiments. To open this bottleneck, kits and reagents will be developed based on Bioo Scientific?s Targeted Transport Technology (T3). Easy-to-use RNAi delivery products will be manufactured, validated and commercialized for use in animal experiments.The broader impacts of this research are twofold. First, researchers will gain ready access to products that greatly simplify the use of RNAi in animals, thereby, stimulating a burst of validation experiments in animals to try to replicate prior results derived from tissue culture experiments. Animals are more complex than their tissue culture counterparts and it is uncertain that results can be duplicated in an animal. Second, T3 has the potential to be used for the therapeutic delivery of RNAi agents. In sum, this project will propel the validation of tissue culture results via T3 enabled animal experimentation, leading to a better understanding of cellular pathways, the identification of novel drug targets, and the potential to deliver RNAi agents as drugs.

该奖项根据 2009 年美国复苏和再投资法案（公法 111-5）提供资金。这个小型企业创新研究 (SBIR) 第二阶段项目将开发优化 RNA 干扰 (RNAi) 在动物中的使用的技术。 RNAi 是表征基因功能的宝贵工具，也是基因治疗的有希望的候选者。 RNAi 在组织培养中的应用已得到充分发展，但在实验动物中的应用有限。 RNAi 药物必须进入细胞才能发挥作用，但这在动物身上已被证明具有挑战性。目前此类技术的缺乏阻碍了大多数重要的 RNAi 动物实验。为了突破这一瓶颈，我们将基于Bioo Scientific的靶向运输技术（T3）开发试剂盒和试剂。易于使用的 RNAi 递送产品将被制造、验证和商业化，用于动物实验。这项研究的更广泛影响是双重的。首先，研究人员将能够方便地获得可大大简化 RNAi 在动物中的使用的产品，从而刺激动物体内的大量验证实验，以尝试复制先前从组织培养实验中获得的结果。动物比组织培养物更复杂，并且不确定结果是否可以在动物中重复。其次，T3 有潜力用于 RNAi 药物的治疗递送。总之，该项目将通过支持 T3 的动物实验推动组织培养结果的验证，从而更好地了解细胞途径、识别新的药物靶点以及将 RNAi 制剂作为药物提供的潜力。