SBIR Phase II: Improved in Vivo Delivery of SiRNA

该奖项是根据2009年美国复苏和再投资法案（公法111-5）资助的。这个小企业创新研究（SBIR）第二阶段项目将开发优化RNA干扰（RNAi）在动物中使用的技术。RNAi是表征基因功能的宝贵工具，并且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很好的发展，但在实验动物中的应用有限。RNAi试剂必须进入细胞才能发挥作用，但这在动物中已被证明具有挑战性。目前缺乏此类技术阻碍了大多数重要的RNAi动物实验。为了打开这一瓶颈，试剂盒和试剂将开发基于生物科学？s有针对性的传输技术（T3）。易于使用的RNAi传递产品将被制造、验证和商业化，用于动物实验。首先，研究人员将获得现成的产品，大大简化了RNAi在动物中的使用，从而刺激了动物验证实验的爆发，试图复制先前来自组织培养实验的结果。动物比其组织培养对应物更复杂，并且不确定结果是否可以在动物中复制。第二，T3具有用于RNAi剂的治疗性递送的潜力。总之，该项目将通过T3启动的动物实验来推动组织培养结果的验证，从而更好地理解细胞通路，识别新的药物靶标，以及将RNAi试剂作为药物递送的潜力。

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.