Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Viral and Non-Viral Approaches for Transient Delivery of mRNA and Proteins

Author(s): Juliane W. Schott, Melanie Galla, Tamaryin Godinho, Christopher Baum, Axel Schambach.

Abstract:

The transient delivery of gene products (RNA or proteins) is not a biotechnological invention but rather an evolutionarily conserved process underlying and regulating a variety of biological functions. On the basis of insights into the underlying mechanisms, several viral and cell-based approaches have been developed for the delivery of RNA or proteins. Prominent applications include the induction of major biological or therapeutic effects on the basis of “hit-and-run” mechanisms, such as vaccination, cell fate modification (reprogramming, differentiation), control of cell trafficking, enhancement of cell regeneration, and genome engineering using sequence-specific recombinases or nucleases. Ideally, procedures for delivery of RNA or proteins should be targeted to specific cells, overcome biophysical hurdles without harming cellular integrity, circumvent the various alarm signals of the innate immune system, allow dose-controlled delivery of functional biomacromolecules, and avoid the induction of an adaptive immune response. Here we review the current state of approaches for the delivery of mRNA and proteins with a focus on RNA viruses, virus-like particles including retrovirus particle-mediated transfer of mRNA or proteins, extracellular vesicles, and cell-penetrating peptides. The basic concepts and recent advances are put into perspective in the context of potential limitations of the technologies and strategies to overcome cellular barriers and defense mechanisms.

Keywords: cell-penetrating peptide, gene therapy, microvesicle, protein transduction, RNA transfer, transient delivery, viral vector, virus-like particle, biomacromolecules, recombinases

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Article Details

VOLUME: 11
ISSUE: 5
Year: 2011
Page: [382 - 398]
Pages: 17
DOI: 10.2174/156652311797415872
Price: $58