The first goal of the proposed project is to investigate expression-targeted gene delivery and derive a model to predict its effectiveness in a variety of situations. This model will provide a better understanding of the technology, which will enable the applicant to rationally design gene therapy constructs that will be of high value to cancer research. The project will then focus upon preliminary applications of expression-targeting. The concept of expression-targeted gene therapy was an incremental advance for transitional cell carcinoma gene therapy, for which the principal investigator has been a leader (patent 20050187177, pending). This transformative concept will be investigated further with the goal of creating a method for cancer screening as well as a potential treatment for transitional cell carcinomas. In a third application of the technology, cells will be engineered to utilize novel signaling cascades. Ancillary benefits of this cellular engineering objective will be the elucidation of basic biologic principles.

The model mentioned above will lead to transfection procedures for which the levels of transgene expression can be controlled. This control will offer a level of variability that will be managed by physiological circumstances within the body. This integration of engineering and life science would advance fundamental knowledge and have a significant impact upon the biomedicine industry. The PI integrates research and teaching and is involved in several outreach programs, including giving K-12 scientific demonstrations to diverse student bodies. A program will be developed to promote community involvement for undergraduate students.

Project Report

The NSF’s CAREER award has been instrumental to the development of our research laboratory and educational endeavors, in addition to furthering the knowledge base supporting expression-targeted gene therapy and applications thereof. Scientifically, work performed as part of the funded project has yielded 7 original science peer-reviewed journal articles to date, with at least 2 more to come. The work has been presented 15 times thus far at conferences and symposia, and an international collaboration was initiated. In terms of scientific training, 3 Ph.D. students were supported in part by the award, and 11 undergraduate students worked on related research projects in the laboratory. Three summers were spent with undergraduate students participating in a program dedicated to minority participation in scientific research, with one student from Grambling appearing as a co-author on a journal publication. Some of the principles uncovered by the laboratory investigations was incorporated into a college course on biotechnology at Tulane University. In terms of scientific achievements that may be applied in a broader context, the project yielded a set of 8 new promoters that we are harnessing to target cancer cells. The targeting is being used for two missions. First, in non-invasive forms of bladder cancer, we are using the technology developed under the CAREER award to target and destroy carcinoma cells. An interesting detail regarding the DNA sequences used for targeting cancer cells was uncovered during the investigations, which ought to allow for personalized gene therapies in the future. Second, in a more systemic application, we are harnessing the targeting technology as an early detection system for various types of cancers. While both of these projects are ongoing, they were initiated and made significant progress via the NSF’s generous award.

Project Start
Project End
Budget Start
2009-07-01
Budget End
2014-06-30
Support Year
Fiscal Year
2008
Total Cost
$400,000
Indirect Cost
Name
Tulane University
Department
Type
DUNS #
City
New Orleans
State
LA
Country
United States
Zip Code
70118