The Institute for Human Gene Therapy was established in 1993 under the direction of Dr. James M. Wilson. Tremendous progress has been made over the last five years in the development of its basic, translational, and clinical research programs. The initial P30 award in gene therapy for Cystic Fibrosis (CF) and genetic diseases provided critical early support to establish an infrastructure, as well as encourage new research initiatives. A fully integrated approach to gene therapy has been developed which is based on fundamental investigation and supported by a number of basic science core laboratories. A translational research program was created to facilitate the transfer of basic discovery to proof-of-concept experiments in humans. This includes a pilot manufacturing facility called the Human Applications Laboratory, a Toxicology Program, a Quality Assurance and Compliance Unit and an effort in regulatory affairs. Twelve tenure track faculty have been recruited over the last five years in the area of CF and genetic diseases. The Institute will increase its total research space from 25,000 to 50,000 net square feet by next Spring, providing additional opportunities for expansion of this program. Tremendous scientific progress has been made in the first term of this grant. Four clinical trials have been started or completed with plans to initiate pilot human experiments in at least three other genetic diseases. This renewal represents a continuation of our initial application. Support is requested for basic science cores including Vector, Cell Morphology, Immunology and Transgenic, as well as the Human Applications Laboratory and Toxicology Program of the Translational Research Program. Our pilot grant program through the NIH and Cystic Fibrosis Foundation has been spectacularly successful; we propose to continue this program in the renewal application. The research base of P30 participants in gene therapy (16 in CF and 37 in genetic diseases. is approximately $26 million (annual direct costs) of which $3.9 million is from NIDDK, $3.7 million is from NHLBI, $3.1 million from NICHD, and $15.2 million from other Federal and non-Federal sponsors. The leadership which has directed the successful implementation of the first phase of this grant will continue in the renewal with Dr. James M. Wilson serving as the Program Director and Dr. John Wolfe serving as the Associate Program Director.
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