GOALI: Gene Delivery: Bioengineering Characterization by Flow Cytometry

许多人类疾病都是由不正常的基因序列引起的。其中一些遗传错误是在出生时遗传的，但许多直到成年才会导致疾病。另一些则是通过与环境接触而获得的。基因治疗是一种广泛的策略，它试图取代或修复这种异常的遗传信息，目的是改变疾病的进程。基因疗法的实际应用一直受到限制，原因有很多，包括创造基因药物的科学挑战和提供这些疗法的临床挑战。拟议的工作通过将学术基础研究和工业应用研究的努力结合起来，以一种新的方式解决这些问题。这一努力的基石是培训下一代工程师--科学家的综合计划。一名攻读生物医学工程博士学位的研究生将在范德比尔特大学的学术实验室研究基因疗法的实施问题。该培训将在Valentis，Inc.(前身为GeneMedicine，Inc.)继续从行业角度进行，Valentis，Inc.是我们这一联合研究和培训计划的合作伙伴。然后，学生将在培训过程中了解基因传递的基本问题和应用问题。学生将在Vanderbilt和Valentis都有一位导师。该学生将返回Vanderbilt大学一段较长的时间，利用从这种分布式培训中获得的广阔视角来开发可能的解决方案。这一领域的另一个进展涉及使用最先进的实验室工具测试这些可能的解决方案，并结合基因传递的数学建模的新想法。在实验室中展示的令人满意的解决方案将由学生在Valentis，Inc.的工业实验室提供的实际约束下进行测试。这项工作的最终目标有两个：(1)一种独特的、有价值的生物技术新培训方法，既适用于学术界，也适用于工业；(2)解决基因交付中的一个重要问题，并为实际应用做好准备。

9902697GiorgioMany human diseases result from abnormal genetic sequences. Some of these genetic errors are inherited at birth, but many do not cause disease until adulthood. Others are acquired though contact with the environment. Gene therapy is the broad strategy that seeks to replace or repair this abnormal genetic information with the goal of altering the course of disease. The practical application of gene therapies has been limited for many reasons including the scientific challenges of creating the 'gene medicines' and the clinical challenges in delivering these therapies.The proposed work addresses these issues in a new way, by combining the efforts of basic research in academic and applied research in industry. The cornerstone of this effort is an integrated plan for training of the next generation of engineer-scientists. A graduate student candidate for the Ph.D. degree in biomedical engineering will study the problem of gene therapy delivery in our academic laboratory at Vanderbilt University. This training will continue from an industrial perspective at Valentis, Inc. (formerly GeneMedicine, Inc.), our partner in this combined research and training program. The student will then understand both the basic and applied problems in gene delivery during this training. The student will have a mentor both at Vanderbilt and at Valentis.This student will return to Vanderbilt University for an extended period to develop possible solutions using the broad perspective gained by this distributed training. Another advance in this field involves the testing of these possible solutions using state-of-the-art laboratory tools coupled with new ideas in mathematical modeling of gene delivery. Satisfactory solutions demonstrated in the laboratory will be tested by the student under the practical constraints offered in the industrial laboratories at Valentis, Inc. There are two final goals of this work: (1) a unique and valuable new training method in biotechnology that uses both academia and industry and (2) a solution to an important problem in gene delivery that is ready for practical application.