Minireview: Delivering the Code: Polyplex Carriers for Deoxyribonucleic Acid and Ribonucleic Acid Interference Therapies

Author:

Christie R. James1,Nishiyama N.23,Kataoka K.1243

Affiliation:

1. Department of Materials Engineering (R.J.C., K.K.), University of Tokyo, Tokyo 113-8656, Japan;

2. Center for Disease Biology and Integrative Medicine (N.N., K.K.), Graduate School of Medicine, University of Tokyo, Tokyo 113-0033, Japan;

3. Graduate School of Engineering and Center for NanoBio Integration (N.N., K.K.), University of Tokyo, Tokyo 113-8656, Japan;

4. Core Research for Evolutional Science and Technology (K.K.), Japan Science and Technology Agency, Saitama 332-0012, Japan

Abstract

Nucleic acid-based therapies offer great potential for treatment of a variety of diseases including cancer by modulating protein expression with DNA or small interfering RNA. However, realization of their full therapeutic potential is currently limited due to an inability to reach the target site in an active form. Identification of delivery barriers such as stability in circulation, resistance to degradation and entrapment in subcellular vesicles has led to development of sophisticated multifunctional synthetic polymers for forming ionic complexes with nucleic acids and also providing performance-enhancing features. The most promising designs comprise features to help increase stability in circulation and also contain functionality to aid in endosome escape of nucleic acid cargo after cellular internalization.

Publisher

The Endocrine Society

Subject

Endocrinology

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