Gene Therapy Of Tay Sachs Using A Modified Adenovirus

3724 Words Dec 3rd, 2014 15 Pages
Gene Therapy of Tay-Sachs Using a Modified Adenovirus
Akash J. Patel

Abstract: Tay Sach’s disease results in an accumulation of lipids in the brain’s nerve cells. The underlying cause of Tay-Sachs disease is a mutation in a gene that codes for a lytic enzyme known as Hexosaminidase A (Hex A). Tay-Sachs as well as other lipid storage diseases are incurable, but we believe that gene therapy has the potential for widespread correction of the lipid storage defect in humans by means of the cross-correction cellular pathway. The vehicle of transportation for the functional copy of the gene is a modified version of the Adenovirus. An advantage of the adenovirus is that its genome does not integrate into the host genome to transcribe its proteins. This allows the adenovirus to express proteins in cells that do not divide, such as neurons. We hypothesize that by infecting neurons with a modified recombinant adenovirus, which encodes a functional copy of the Hex A gene, we can enhance functional enzyme expression. Through an intrathecal injection and an injection in the ventral tegmental area of the patient we hope to see a distribution of the vector through axonal transport. We also hope to see a distribution of the enzyme through the neurons’ secretion-uptake pathway. Using magnetic resonance imaging we will track the progression of the disease. Similar studies involving lipid storage diseases in mice have shown that gene therapy has significantly lessened symptoms and lowered…

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