GENE THERAPY RESOURCE PROGRAM (GTRP) Header
GENE THERAPY RESOURCE PROGRAM (GTRP) Header

WHAT IS THE NHLBI GENE THERAPY RESOURCE PROGRAM?

The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions.  The GTRP provides resources for gene therapy research primarily in heart, lung, and blood diseases as reflected in the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm).  Requests for resources for gene therapy research that are consistent with the missions of other NIH Institutes may also be considered by the Program.

Resources are provided in the form of preclinical and clinical-grade vector production, pharmacology/toxicology testing, immunology testing, clinical trials funding assistance, and regulatory support at no cost to the investigator.  Investigators must first receive approval of their Registration with the Program in order to request resources.

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.

University of
Pennsylvania


Produces large-
and small-scale
viral and non-viral vectors for studies in basic research directed toward clinical applications; performs immunology testing

The Children’s Hospital of Philadelphia

Produces scalable
clinical-grade adeno-
associated virus (AAV) vectors for use in clinical studies and GMP process-comparable AAV vectors for use in pharm/tox studies

Indiana
University


Produces scalable
clinical-grade lentiviral vectors for use in clinical studies and GMP process-comparable lentivirus vectors for use in pharm/tox studies

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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  » Article - April 2009
»Publications and Presentations
  by GTRP Investigators