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Review
. 2019 Oct:217:119291.
doi: 10.1016/j.biomaterials.2019.119291. Epub 2019 Jun 20.

RNA delivery biomaterials for the treatment of genetic and rare diseases

Weiyu Zhao  1 , Xucheng Hou  1 , Olivia G Vick  2 , Yizhou Dong  3
Affiliations
Review

RNA delivery biomaterials for the treatment of genetic and rare diseases

Weiyu Zhao et al. Biomaterials. 2019 Oct.

Abstract

Genetic and rare diseases (GARDs) affect more than 350 million patients worldwide and remain a significant challenge in the clinic. Hence, continuous efforts have been made to bridge the significant gap between the supply and demand of effective treatments for GARDs. Recent decades have witnessed the impressive progress in the fight against GARDs, with an improved understanding of the genetic origins of rare diseases and the rapid development in gene therapy providing a new avenue for GARD therapy. RNA-based therapeutics, such as RNA interference (RNAi), messenger RNA (mRNA) and RNA-involved genome editing technologies, demonstrate great potential as a therapy tool for treating genetic associated rare diseases. In the meantime, a variety of RNA delivery vehicles were established for boosting the widespread applications of RNA therapeutics. Among all the RNA delivery platforms which enable the systemic applications of RNAs, non-viral RNA delivery biomaterials display superior properties and a few biomaterials have been successfully exploited for achieving the RNA-based gene therapies on GARDs. In this review article, we focus on recent advances in the development of novel biomaterials for delivery of RNA-based therapeutics and highlight their applications to treat GARDs.

Keywords: CRISPR; Genetic and rare diseases (GARDs); RNA delivery biomaterials; RNA therapeutics; RNAi; mRNA.

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Conflict of interest statement

Conflicts of interest

The authors have no competing interests to declare.

Figures

Figure 1.
Figure 1.
A representation of genetic and rare diseases (GARDs) that have been explored using non-viral biomaterials based RNA therapeutics.
Figure 2.
Figure 2.
An illustration of representative non-viral biomaterials (outer circle) together with versatile RNA therapeutics (inner circle) that have been exploited for treating genetic and rare diseases (GARDs). [, –49]
Figure 3.
Figure 3.
A list of biomaterials and RNAs that have been explored for the treatment of different GARDs.
See this image and copyright information in PMC

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