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 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.

University of
Pennsylvania


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

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

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

HIGHLIGHTS
RSA Submissions are Accepted on a Continuous Basis

»
 Instructions for Submitting
  an RSA

» RSA Review Process
INFORMATION CENTER

» Regulatory Guidelines

» GTRP Presentations at the
  ASGT Meeting May 28, 2008

» NHLBI Home