Gene Therapy Resource Program Pharmacology-Toxicology Core Lab

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The objective of the NHLBI Gene Therapy Resource Program (GTRP) renewal is to continue supporting the translation of basic research in gene therapy to clinical application in the areas of heart, lung, and blood diseases. The GTRP renewal will continue to provide 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 renewal will provide funds and regulatory assistance to investigators wishing to conduct gene therapy clinical trials. The GTRP renewal will consist of the following five components: 1) a clinical coordinating center that will oversee and coordinate the logistics of the core laboratories and provide regulatory and funding assistance for clinical trials; 2) a preclinical-grade vector production core laboratory (PCL); 3) a clinical-grade vector production core laboratory for adeno-associated virus (AAV); 4) a clinical-grade vector production core laboratory for lentivirus (LCL); and 5) a pharmacology/toxicology core laboratory (PTCL).