? Core B: Vector Core Recombinant adeno-associated virus (rAAV) is a popular gene transfer platform technology to deliver transgenes for in vivo gene therapy. Our recent discovery of a diverse family of novel primate AAVs that are capable of transducing a variety of target tissues much more robustly than the first generation of AAV2-based vectors remarkably expanded the utility of rAAVs in different gene therapy applications including treatment of alpha-1 antitrypsin deficiency (AATD). The Viral Vector Core (Core B) is the central component of our collaborative effort to develop new approaches to gene therapy for AATD. The main objectives of Core B are to provide the investigators of other subprojects and Cores with high quality of preclinical grade rAAV vectors to address their specific scientific questions in a variety of animal models as well as reliable and reproducible AAV neutralizing antibody testing service. Core B will rely on the resources and experience of the University of Massachusetts Viral Vector Core (Guangping Gao, Director), an internationally well recognized vector service with abundant experience in vector production, quality control, and uses in different in vivo gene transfer applications to accomplish its goal through the following Aims:
Aim 1. To design, create, produce, purify and quality control test preclinical grade rAAV vector preparations for all subprojects and cores of this tPPG, and Aim 2. To perform in vitro and in vivo neutralizing antibody assays to screen nonhuman primate animals for studies proposed by subproject 2 and study seroepidemiology of novel AAV capsids developed by subproject 4 in human populations.
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