Illinois 2011 NanoBiophotonics Summer School: Dr. Naash - Motivation
University of Illinois at Urbana-Champaign
Dr. Muna Naash, University of Oklahoma Professor, Department of Cell Biology Director, Graduate Program
EDUCATION Ph.D., Biochemistry/Cell Biology, Baylor College of Medicine, Houston, Texas
RESEARCH SUMMARY The goal of our program is to advance our current compacted DNA nanoparticle based gene therapy technology to enable efficient and long-lasting gene delivery to dividing and non-dividing cells. The program merges experts with molecular bioengineering, physics, chemistry, and computer science backgrounds at OUHSC, Stanford University and Copernicus Therapeutics, Inc, to accelerate essential preclinical steps for effective non-viral gene therapy. We have filed two patents for the application of this technology in ocular diseases and have shown in our recent publication the efficacy of this technology in transfecting ocular tissues (Farjo, et al, 2006). Our goal is to engineer DNA vectors with efficient uptake and transport through the plasma membrane (PM) that can provide persistent transgene expression without toxicity. This technology can unimolecularly compact DNA with lysine polymers substituted with polyethylene glycol (PEG) into neutral charge nanoparticles with radii of less than 8 nm. These particles can penetrate the cell membrane via nucleolin receptor associated endocytosis and cross the nuclear membrane pore to the nucleus within 15 minutes. The DNA condensation formulation can compact linear or circular DNA and RNA which enabling us to eliminate plasmid backbone sequences known to play a significant role in inhibiting gene expression. The potential scientific and clinical benefits of these enhancements are substantial.
Assistant Crew: Abby Sobh Obaid Sarvana
SCHOOL ORGANIZERS: 2011 Gabriel Popescu, Stephen Boppart, Marina Marjanovic, Rohit Bhargava, Logan Liu, Kimani Toussaint, Scott Caney, Peter Wang, Samir Sayegh , Nahil Sobh
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