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GENE THERAPY FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS USING CHITOSAN-BASED NANOPARTICLES AS NONVIRAL GENE DELIVERY VECTORS [Author names removed] Abstract In recent years, nanoparticles have been utilized in the development of gene therapy for the treatment of lysosomal storage diseases; however, problems remain in targeting the particles to specific cells in the body. 1 Of late, scientists have used chitosan-based hydrogel nanoparticles for drug delivery. Nanogels are stimuli dependent on pH and temperature, which allows them to be targeted to cells with a specific pH or temperature. 2 This project will utilize the chitosan- nanogels for gene delivery and compare the results to the currently used organically modified silica nanoparticle (ORMOSIL) as the control. 3 The proposed project will answer the following questions. Can the hydrogel complex deliver the chitosan nanoparticle to the cell? Will the chitosan nanoparticle transport the gene to the nucleus? Will the gene be incorporated into the genome? The successful outcomes will allow more work to be done with the stimuli responsive hydrogels, and thus, revolutionize gene therapy. Objective This project proposes to deliver DNA that codes for production of hexosaminidase A, a key protein involved in lysosomal storage disorders, using a chitosan nanoparticle, in hopes of transporting the gene to the nucleus and incorporating it into the genomes of target cells. The control to which we will be comparing the efficiency of this method is ORMOSIL, a currently used nanoparticle. 4 Introduction In all eukaryotic cells, there are organelles called lysosomes, where the degradation or digestion of waste products, fats, carbohydrates, proteins, and other macromolecules takes place with the help of hydrolytic enzymes. Due to genetic defects, some eukaryotic cells experience lysosomal storage disorders (LSDs), which involve a buildup of undigested material within lysosomes that leads to the enlargement of lysosomes and eventually to cell degeneration. This is a very rare disease, but a few known LSDs are Tay-Sachs, Hunter syndrome, and Sly disease. In Tay-Sachs disease, for example, GM2 gangliosides build up in the cell, resulting in damage to nerve cells, especially in the brain. 5 Hexosaminidase A is a protein that helps break down these gangliosides, so it is the enzyme we will be replacing. All LSDs cause some form of physical and mental degradation, causing death. The current method for treatment of Lysosomal Storage Disorders involves several types of prescription medication, respiratory care, and physical therapy. Gene therapy is a promising new possibility of treatment that will give better results in the future. 6
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This note was uploaded on 01/06/2012 for the course UGS 303 taught by Professor Foster during the Fall '08 term at University of Texas.

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