The Administrative Core will be based at The Wistar Institute in Dr. H. Ertl's laboratory/office complex. It will provide administrative and logistic support and budgetary oversight for the different scientific projects. The primary aim of the Administrative Core is to manage and oversee the scientific efforts of the Projects. To this end, the Core will organize regular meetings ofthe investigators and their collaborators. In addition, the Core will arrange for meetings of the Executive Committee and the annual meeting of the External Scientific Advisory Board (ESAB). The Administrative Core will also be in charge of producing and submitting the annual progress report to NHLBI. The Administrative Core will provide budgetary oversight over the program. The Administrative Core will be in charge to obtain and maintain 'umbrella'lACUC and IRB approvals to cover the scientific efforts of the program. The Administrative Core will arrange for the resolution of potential conflicts;depending on the type of conflict this may further involve NHLBI and members ofthe ESAB. In case the PI of this program becomes unable to fulfill her obligations to this program, the Administrative Core will arrange for a meeting ofthe Executive Committee with representatives from NHLBI and the ESAB to decide upon a new PI in a timely manner. The Administrative Core will facilitate sharing of reagents by assisting in speedy execution of material transfer agreements
Gene replacement therapy has the potential to cure inherited diseases. Viral vectors such as AAV vectors are highly suited to deliver genes, but, nevertheless, may not result in sustained transgene expression in humans, as antigens ofthe viral vector and a potentially foreign transgene product may elicit adaptive immune responses that cause rejection of vector-transduced cells. Understanding such immune responses is a pre-requisite to devise avenues to circumvent them. This in turn may then allow for sustained AAV-mediated gene transfer and the associated benefits to human subjects suffering from genetic diseases.
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