Gene Therapy And Neuroprotection

我们的实验室现在生产用于基因传递的腺相关病毒（AAV）载体。我们已经获得了包装7种不同血清型AAV载体所需的试剂，从而在接受我们感兴趣的转基因的细胞/组织类型中提供了更多选择。这些载体的一个用途是将基因传递到培养的神经元中。我们目前正在描述不同血清型在原代神经元培养中的嗜性和毒性。这些研究将建立适当的血清型和MOI，用于转导我们感兴趣的基因。利用这些感染参数，我们可以将各种基因传递到甲基苯丙胺毒性、兴奋性毒性、缺氧和帕金森症的体外模型中。以表征骨形态发生蛋白（BMPs）保护和再生特性的信号转导途径。我们的第二种方法是利用AAV载体在中风、帕金森？S疾病和甲基苯丙胺毒性具体来说，我们评估了BMP7及其信号分子的保护和再生特性。目前，我们正在测试AAVBMP7和AAVSMADs在帕金森病模型中的作用。S和甲基苯丙胺毒性。

Our laboratory now produces adeno-associated viral (AAV) vectors for gene delivery. We have obtained the reagents needed to package 7 different serotypes of AAV vectors thereby offering more selection in the type of cells/tissues that receive our transgene of interest. One use of these vectors is to delivery genes to neurons in culture. We are currently characterizing the tropism and toxicity of the various serotypes in primary neuronal cultures. These studies will establish the appropriate serotype and MOI to use to transduce our genes of interest. Using these infection parameters we can deliver various genes to in vitro models of methamphetamine-toxicity, excitotoxicity, hypoxia and Parkinson?s disease to characterize the signal transduction pathways involved in the protective and regenerative properties of bone morphogenetic proteins (BMPs). Our second approach uses AAV vectors for in vivo delivery to the brain in models of stroke, Parkinson?s disease and methamphetamine-toxicity. Specifically, we evaluate the protective and regenerative properties of BMP7 and its signaling molecules. Currently, we are testing the role of AAVBMP7 and AAVSMADs in models of Parkinson?s and methamphetamine-toxicity.