The overall necessity of this facility is to provide a laboratory solely dedicated to the development of clinical applications of gene therapy. The faculty will be regulated by the FDA and required to practice both Good Laboratory Practices (GLP) as well as Good Manufacturing Practices (GMP). It will also be certified to operate as a BL2 containment facility in the handling of potential biohazardous material.
Specific aims are described below: A) To provide a state-of-the-art laboratory with BL2 containment as well as GLP and GMP guidelines, B) To provide facilities and expertise in the development of gene transfer substrates that are being reviewed and certified by the FDA, C) To provide facilities and expertise in the harvest, isolation and cultivation of human cells that will be genetically modified and used as either preclinical data in the documentation of the feasibility and safety of gene transfer or used in an actual clinical protocol, D) To provide facilities and expertise in the implementation of preclinical studies under conditions that are acceptable to the FDA, E) To establish protocols and standard operating procedures (SOPs) for the applications of human gene therapies of general use to the institution, F) To establish a series of standard quality controls based on SOPs that are acceptable to the FDA that can be utilized in the certification of protocols.
|Bals, R; Weiner, D J; Meegalla, R L et al. (2001) Salt-independent abnormality of antimicrobial activity in cystic fibrosis airway surface fluid. Am J Respir Cell Mol Biol 25:21-5|
|Gao, G P; Engdahl, R K; Wilson, J M (2000) A cell line for high-yield production of E1-deleted adenovirus vectors without the emergence of replication-competent virus. Hum Gene Ther 11:213-9|
|Chirmule, N; Propert, K; Magosin, S et al. (1999) Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 6:1574-83|
|Zuckerman, J B; Robinson, C B; McCoy, K S et al. (1999) A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther 10:2973-85|
|Bals, R; Weiner, D J; Moscioni, A D et al. (1999) Augmentation of innate host defense by expression of a cathelicidin antimicrobial peptide. Infect Immun 67:6084-9|
|Chirmule, N; Truneh, A; Haecker, S E et al. (1999) Repeated administration of adenoviral vectors in lungs of human CD4 transgenic mice treated with a nondepleting CD4 antibody. J Immunol 163:448-55|
|Jiang, Q; Mak, D; Devidas, S et al. (1998) Cystic fibrosis transmembrane conductance regulator-associated ATP release is controlled by a chloride sensor. J Cell Biol 143:645-57|
|Jooss, K; Turka, L A; Wilson, J M (1998) Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig. Gene Ther 5:309-19|
|Chirmule, N; Hughes, J V; Gao, G P et al. (1998) Role of E4 in eliciting CD4 T-cell and B-cell responses to adenovirus vectors delivered to murine and nonhuman primate lungs. J Virol 72:6138-45|
|Goldman, M J; Lee, P S; Yang, J S et al. (1997) Lentiviral vectors for gene therapy of cystic fibrosis. Hum Gene Ther 8:2261-8|
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