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Gene Therapy for Neurological Disorders
Release Date: July 19, 2001
Announcement Number: RFA-NS-02-007
Application Receipt Date:
November 16, 2001
Letter of Intent Date: October 15, 2001
Funding Contact: Robert Finkelstein, Ph.D.
Program Area: Neurogenetics
Brief Description:
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, more studies addressing the toxicity of specific vector/transgene
combinations, and greater knowledge of the long term effects of expressing specific transgenes in the nervous system. This
RFA is intended to encourage research projects that address these or related translational issues in the context of specific
neurological disorders.
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