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。这个小型企业创新研究(SBIR)第二阶段项目将开发在动物身上优化使用RNA干扰(RNAi)的技术。RNAi是表征基因功能的无价工具，也是基因治疗的一个有前途的候选者。RNAi在组织培养中的应用已经很成熟，但在实验动物中的使用有限。RNAi试剂必须进入细胞才能发挥其作用，但这在动物身上已被证明是具有挑战性的。目前此类技术的缺乏阻碍了大多数重要的RNAi动物实验。为了打开这一瓶颈，将基于生物科学？S靶向传输技术(T3)开发试剂盒和试剂。易于使用的RNAi递送产品将被制造、验证并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，研究人员将获得可以大大简化RNAi在动物身上使用的产品，从而刺激动物身上的一系列验证实验，试图复制组织培养实验得出的先前结果。动物比它们的组织培养对手更复杂，结果能否在动物身上复制还不确定。第二，T3有可能用于RNAi制剂的治疗。总而言之，该项目将通过启用T3的动物实验来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，并将RNAi试剂作为药物输送的可能性。

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.