The primary responsibility of the Vector Core (Core B) is the resolution of administrative, fiscal, and scientific issues pertaining to recombinant vector production.
The Specific Aims of the Vector Core are central to the overall success of this proposal, and include: 1. Production, purification, and characterization of research grade recombinant adeno-associated (AAV) vector stocks for research projects: a. listed in this proposal and, b. non-proposed related, peer reviewed, NIH funded projects focusing upon heart, lung or blood diseases. 2. Process developed of methods of production and purification of AAV vectors and assays for characterization. 3. cGMP manufacturing and QC release of rAAV vectors for use in preclinical studies. 4. Promotion of cohesive, continuing scientific interactions with all project investigators so that the latest technological advances in vector design or development can be implemented. It is anticipated the production of high titer, qualified stocks of recombinant adenovirus and rAAV vectors will expedite acquisition of preclinical gene transfer data. Furthermore, use of cells and virus or vector stocks that are free of mycoplasma, helper virus (adenovirus) and particularly wild type AAV, will remove confounding variables which have plagued interpretation of several gene transfer studies. Finally, the Vector Core is not envisioned to be a static entity, but will interact with investigators and fully participate in the implementation of the latest technologic advances to improve gene transfer.
| Paz, Helicia; Wong, Christie A; Li, Wei et al. (2007) Quiescent subpopulations of human CD34-positive hematopoietic stem cells are preferred targets for stable recombinant adeno-associated virus type 2 transduction. Hum Gene Ther 18:614-26 |
| Santat, Leah; Paz, Helicia; Wong, Christie et al. (2005) Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice. Proc Natl Acad Sci U S A 102:11053-8 |
| Smejkal, Gary B; Shainoff, John R; Yakubenko, Anna V (2002) Direct chemiluminescent immunodetection of proteins in agarose gels. Electrophoresis 23:979-84 |
