Projects
Specially Promoted Research, Grant-in-Aid for Scientific Research,
Japan Society for the Promotion of Science
Title of the Project: Development of polymeric micelles for brain-targeted delivery of nucleic acid drugs to treat intractable neurological diseases
Reseach Term: FY2013 - FY2017
Principal Investigator: Kazunori Kataoka
Purpose and Background of the Research
In the aging society of developed countries, strong demand is placed on the cure of neurological disorders, such as Alzheimer's disease, which now affects over 200,000 people in Japan. Recent progress in understanding the underlying molecular mechanisms of neurological disorders holds promise for development of nucleic acid-based therapies that modulate disease pathways through regulation of gene expression. To this end, novel drug delivery systems (DDS) are indispensable for the successful function of these nucleic acid drugs in brain cells, such as neurons, by penetrating the tight barrier of endothelial/ependymal cell layers that protect the brain from outer environments.
In this context, this research project aims to develop nano DDS that overcome the brain barrier for nucleic acid delivery to central nervous system, and further, demonstrate the efficacy of the nano DDS on the molecular therapy of neurological disorders. In particular, multifunctional block copolymers are elaborated to construct polymeric micelles featuring biocompatibility, target specificity, and stimuli responsivity for smart nucleic acid delivery (Figure 1).
