Gene Therapy Using Nanoparticles:

 (In collaboration with Dr. Michal Stachowiak at UB south Campus and Prof. Savio Woo in Mount SinaiSchool of Medicine, New York)

Gene therapy is a collective term which is given to numerous possibilities that can be brought about by manipulation at the genetic level. Apart from generating superheroes like spiderman and ninja turtles, more realistic aspirations of gene therapy include (a) correction of diseases related to single-gene malfunctions (like cystic fibrosis) by replacing the defunct gene with functional ones, (b) silencing of malevolent genes like the cancer causing oncogenes and (c) development of transgenic animal models of various diseases which would help accelerate the search for their cure.

Nanoparticles play a major role in this gene transfer as unlike viruses, the natural gene transporters, they are not associated with toxic side-effects. In our laboratory, we have developed cationically modified ORMOSIL nanoparticles that can bind and protect DNA and efficiently transfer genes across a number of cell lines, while displaying minimal toxicity. In animal studies, we have found that these gene doped nanoparticles can robustly transfect cells in liver (hepatocytes) and the central nervous system (CNS), as seen by the expression of the reporter gene encoding the green fluorescent protein (Fig. 2). At present, using the nanoparticles we are investigating the effect of therapeutic gene transfer on experimental animals carrying various disorders of the CNS and liver like Parkinson’s disease and phenylketonurea.