"Technology for Targeted Genetic Therapies"
James Dahlman, Ph.D.
Georgia Tech & Emory University
Regardless of their biological mechanism of action, all DNA and RNA therapies are limited by one problem: drug delivery. DNA or RNA must be avoid clearance by the immune system, kidney, and spleen, access the target cell in a complex microenvironment, and enter the cytoplasm. Engineers and chemists have designed thousands of chemically distinct nanoparticles to target RNA or DNA. However, after they are synthesized, the expensive nature of animal experiments forces us to study whether the nanoparticles work in cell lines, even though drug delivery in cell culture does not recapitulate drug delivery in a living animal.
To expand the number of nanoparticles we could study in vivo, we designed a high throughput nanoparticle / DNA barcoding platform to study many nanoparticles in a single animal. We used a high throughput microfluidic device that produces small, stable nanoparticles; each nanoparticle was formulated to carry a distinct DNA barcode. This new barcoding provides robust, linear, and repeatable results in vivo, and generates data that matches functional delivery of genetic therapies. We will describe this new in vivo screening platform, and discuss its applications to targeted anti-cancer gene therapies.
Integrated Cancer Research Center
Georgia Tech has been a leader in the development of collaborative approaches to both cancer diagnostics and therapeutics. The mission of the Integrated Cancer Research Center (ICRC) is to facilitate integration of the diversity of technological, computational, scientific and medical expertise at Georgia Tech and partner institutions in a coordinated effort to develop improved cancer diagnostics and therapeutics.