Research Question
Research Problem
With current methods of gene expression alteration, many cells are harmed to provide small amounts of results. For example, electroporation is a method where the cell is opened with electricity. Such malignant methods compounded with inefficiency drove us to find an alternate way to provide gene expression alteration.
How can therapeutic small RNAs be efficiently and specifically delivered into primary cells?
Electroporation. http://i.imgur.com/MaR3ubp.jpg
Gene therapy is a promising new technology that has the potential to treat virtually all genetic diseases and facilitate research for cures. However, conventional methods of gene therapy are limited because they are extremely harmful to cells, and thus can only be used on immortalized cell lines (Yao & Eriksson, 2000). Furthermore, these methods lack specificity, targeting all cells without regard for cell type (Sharma & Sharma, 1997). A novel method for delivering silencing RNA (siRNA) to alter cellular gene expression was recently developed at the NIH. This method utilizes a modular system consisting of a specific ligand coupled to a Hepatitis B Virus-derived RNA binding domain. This enables researchers to deliver siRNAs to specific cell types through cell-specific receptor/ligand interactions. Because this technique is so practical, it can be applied to virtually any disease or assist research for these diseases. The method is also gentle enough to alter gene expression on primary cells more efficiently than conventional methods and targets specific cells. Our project aims to produce a modular siRNA delivery vehicle protein, expose specific cells to the delivery vehicle coupled to siRNAs, and quantify specific changes in gene expression.
Abstract
