The Clinical Coordinating Center (CCC) for the existing Gene Therapy Resource Program (GTRP) facilitates the translation of basic research in gene therapy to clinical application in the areas of heart, lung, and blood diseases. The GTRP renewal allows the program to continue providing resources in the areas of preclinical and clinical-grade vector production in accordance with Good Manufacturing Practices (GMP), and in the conduct of pharmacology/toxicology studies. In addition, the GTRP will continue to provide funds and regulatory assistance to investigators wishing to conduct gene therapy clinical trials. The GTRP consist of a clinical coordinating center that oversees and coordinates the logistics of the core laboratories and provides regulatory assistance for clinical trials;a preclinical-grade vector production core laboratory;a clinical-grade vector production core laboratory for adeno-associated virus (AAV);a clinical-grade vector production core laboratory for lentivirus;and a pharmacology/toxicology core laboratory.
