Literature DB >> 31689462

Polymeric siRNA gene delivery - transfection efficiency versus cytotoxicity.

Anna Kargaard1, Joost P G Sluijter2, Bert Klumperman3.   

Abstract

Within the field of gene therapy, there is a considerable need for the development of non-viral vectors that are able to compete with the efficiency obtained by viral vectors, while maintaining a good toxicity profile and not inducing an immune response within the body. While there have been many reports of possible polymeric delivery systems, few of these systems have been successful in the clinical setting due to toxicity, systemic instability or gene regulation inefficiency, predominantly due to poor endosomal escape and cytoplasmic release. The objective of this review is to provide an overview of previously published polymeric non-coding RNA and, to a lesser degree, oligo-DNA delivery systems with emphasis on their positive and negative attributes, in order to provide insight in the numerous hurdles that still limit the success of gene therapy.
Copyright © 2019 Elsevier B.V. All rights reserved.

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Year:  2019        PMID: 31689462     DOI: 10.1016/j.jconrel.2019.10.046

Source DB:  PubMed          Journal:  J Control Release        ISSN: 0168-3659            Impact factor:   9.776


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