CORE--VECTOR

载体核心(核心B)的主要责任是解决与重组载体生产有关的行政、财政和科学问题。载体核心的具体目标是这项提议总体成功的关键，包括：1.研究项目的研究级重组腺相关(AAV)载体库存的生产、纯化和鉴定：a.本提议中列出的；b.非提议的、同行评审的、由NIH资助的专注于心脏、肺或血液疾病的项目。2.发展了AAV载体的生产和纯化方法以及用于表征的检测方法。3.临床前研究用重组腺相关病毒载体cGMP的制备和质量控制释放。4.促进与所有项目调查人员的连贯、持续的科学互动，以便能够实施媒介设计或开发方面的最新技术进步。预计高滴度、合格的重组腺病毒和rAAV载体的生产将加快临床前基因转移数据的获取。此外，使用不含支原体、辅助病毒(腺病毒)和特别是野生型AAV的细胞和病毒或载体，将消除困扰几项基因转移研究解释的混杂变量。最后，媒介核心并不是一个静态的实体，而是将与调查人员互动，并全面参与实施最新的技术进步，以改善基因转移。

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.