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Purpose: The goal of this Request for Applications (RFA) is to accelerate the translation of gene transfer methodologies into the clinic. Gene therapy holds tremendous promise for the treatment of neurological disorders. Despite recent advances in this area however, specific scientific, technological, and safety goals must be achieved before gene transfer becomes a viable therapeutic alternative. Current needs include more effective methods for controlling the expression of therapeutic transgenes in the brain, better strategies for vector delivery and monitoring transgene expression, …
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