GOALI: Gene Delivery: Bioengineering Characterization by Flow Cytometry

点击翻译按钮获取中文摘要

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.