Topic Introduction

An Overview of Condensing and Noncondensing Polymeric Systems for Gene Delivery

Adapted from "An Overview of Condensing and Noncondensing Polymeric Systems for Gene Delivery," Chapter 34, in Gene Transfer: Delivery and Expression of DNA and RNA (eds. Friedmann and Rossi). Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY, USA, 2007.

INTRODUCTION

Self-assembling synthetic vectors for DNA delivery are designed to fulfill several biological functions. They must be able to deliver their genetic payload specifically to the target tissue/cells in a site-specific manner, while protecting the genetic material from degradation by metabolic or immune pathways. Furthermore, they must exhibit minimal toxicity and be proven safe enough for therapeutic use. Ultimately, they must have the capability to express a therapeutic gene for a finite period of time in an appropriate, regulated fashion. The DNA encapsulated in these vectors may be in a condensed or noncondensed form, depending on the nature of the polymer and the technique used for formulating the vector system. The whole process presents many barriers at both tissue and cellular levels. Overcoming these hurdles is the principal objective for efficient polymer-based DNA therapeutics.