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.
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