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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 ( 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.

Preclinical Vector Core

University of Pennsylvania

Produces large-, routine, and small-scale viral and non-viral based vectors for studies in basic research directed toward clinical applications and provides immunology services

GTRP page

Clinical-Grade AAV Vector Core

The Children’s Hospital of Philadelphia

Produces scalable clinical-grade adeno-associated virus (AAV) vectors for use in clinical studies; GMP process-comparable AAV vectors for use in pharmacology/ toxicology studies

GTRP page

Clinical-Grade Lentivirus Vector Core

Indiana University

Produces scalable clinical-grade lentiviral vectors for use in clinical studies; produces GMP process-comparable lentivirus vectors for use in pharmacology/ toxicology studies

GTRP page

Pharmacology/ Toxicology Core

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

GTRP page

Clinical Coordinating Center

Social & Scientific Systems, Inc.

Coordinates RSA process management, Steering Committee management, regulatory support, and clinical trials funding assistance

GTRP page