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Gene Transfer and Rare Diseases Workshop

Thursday, September 13, 2012
Rockville Hilton, Rockville, Maryland

The NIH Office of Biotechnology Activities and Office of Rare Disease Research, NCATS, are co-sponsoring a workshop to discuss the advances in and challenges of gene transfer for rare diseases. There have been recent promising clinical successes in gene transfer protocols for rare diseases, such as retinal degeneration, hemophilia, and immune disorders. Investigators will discuss their results to date and opportunities and challenges for extending these successes to other similar rare diseases. The panel discussions will focus on strategies for maximizing opportunities for data sharing or platform development.

PJ Brooks, Ph.D., (301) 443-0513

Co-funding Institute(s):
Office of the Director
Office of Rare Diseases Research

From the National Center for Advancing Translational Sciences

From the National Institutes of Health

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