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The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions for heart, lung, and blood diseases.
The GTRP provides resources in the form of preclinical and clinical-grade vector production, pharmacology/toxicology testing, clinical trials funding assistance, and regulatory support at no cost to the investigator. Resources are provided to investigators conducting research in gene therapy for heart, lung, or blood diseases that is consistent with the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm), and who have received approval of their Registration application for the Program.
The GTRP, directed by the NHLBI Gene Therapy Group, consists of three vector production cores, a pharmacology/toxicology testing core, and a clinical coordinating center. A Scientific Review Board and Steering Committee review Request for Service Applications and make recommendations to the NHLBI Gene Therapy Group regarding the applications’ scientific merit, feasibility, and compatibility with the program’s mission.
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University of
Pennsylvania
Produces large-
and
small-scale
viral and
non-viral vectors for studies in basic research directed toward clinical applications
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The Children’s Hospital
of Philadelphia
Produces scalable
clinical-grade adeno-
associated virus (AAV)
vectors for use in heart, lung, and blood clinical studies |
Indiana
University
Produces scalable
clinical-grade lentiviral
vectors for use in heart, lung, and blood clinical studies
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Lovelace Biomedical
and Environmental
Research Institute
Performs toxicology testing and
bio-distribution studies of
vectors in large and small
animal models as a prerequisite for use in clinical studies |
Social & Scientific
Systems, Inc.
Coordinates
RSA process management, Steering
Committee management,
regulatory support,
and clinical trials
funding
assistance
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