The Institute for Human Gene Therapy (IHGT) at the UP provides an academic-based infrastructure for the development of successful gene therapies from basic vector design to proof-of-concept in human pilot experiments. Basic and clinical research programs in gene therapy are focused on specific diseases through the efforts of individual faculty supported through investigator-initiated grants. In addition, the IHGT has developed unique resources within academia to facilitate the translation of basic research to pilot human experiments. The program in vector developments and production focuses on innovations in vector design, as well as production of vectors for applications to humans through the HAL. Preclinical testing of novel vectors is performed under the auspices of the program in Animal Resources and Toxicology (ARAT). This application requests support for the HAL in the context of the NGVL Program. The HAL at the UP is a 3,000 net sq. ft. laboratory that operates in accordance with Food and Drug Administration (FDA) guidelines of Good Manufacturing Practices (GMP) and Good Laboratory Practices (GLP), and is equipped to work with biohazardous material at the BL2+ level. The facility contains five independent manufacturing sites each capable of producing clinical grade recombinant viruses and/or manipulating human cells for use in clinical trials. The HAL was the first academic or commercial facility to successfully bring both retroviral and adenoviral technology through Investigational New Drug (IND) approval. The laboratory has established a Transgene Diagnostic Program that is capable of detecting and quantifying recombinant sequences in biological samples. This application requests support for the basic infrastructure of HAL and costs to produce and certify clinical grade viruses. The laboratory is currently supporting, or has made commitments to support, 27 clinical project from nine different institutions. The projects involve a diverse group of diseases, including inherited dyslipidemias, cystic fibrosis, lethal pediatric metabolic diseases and cancer.
| Raper, Steven E; Yudkoff, Marc; Chirmule, Narendra et al. (2002) A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther 13:163-75 |
| Auricchio, A; Hildinger, M; O'Connor, E et al. (2001) Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum Gene Ther 12:71-6 |
