Gene therapy and enzyme replacement for Batten disease

University Of Iowa, Iowa City IA

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Linked publications & trials

Abstract

[unreadable] DESCRIPTION (provided by applicant): This proposal is focused on gene therapy approaches for lysosomal storage diseases affecting the D central nervous system. Using late infantile neuronal ceroid lipofuscinosis (LINCL / Batten Disease) as a D model disease, I propose to test hypotheses regarding delivery of a gene to the CNS of a murine disease D model. Recent data from our lab shows that adeno-associated virus type 4 (AAV4) is effective in directing D widespread distribution of a lysosomal enzyme in the murine brain, which is an exciting finding given that D LINCL patients exhibit widespread CNS pathology. The ability of AAV4 to direct gene transfer in a mouse D model of LINCL will be tested here. In addition, I propose to develop a tetracycline-regulated viral vector as I clinical applications of gene transfer will likely require regulated expression to minimize immune responses C or other adverse effects. A regulated vector will also allow me to address the duration of benefits after D cessation of enzyme expression. [unreadable] [unreadable] [unreadable]