Rethinking Delivery: Viral Vectors and RNA Interference
Journal Title: Journal of RNA and Genomics - Year 2017, Vol 13, Issue 1
Abstract
Efficient delivery of nucleic acids to their intended target cells remains the major complication holding back the widespread adoption of RNA interference (RNAi) therapies. In recent years, substantial progress has been made in the development of delivery systems for small interfering RNAs (siRNAs), including lipid formulations, nanoparticles and functional groups covalently coupled to siRNAs. So far the vast majority of clinical trials involving RNAi therapies have relied on the use of such chemically generated systems. Some organs, however, remain difficult to target with currently available delivery agents. Viral vectors are a promising alternative to deliver short hairpin RNA (shRNA) expression cassettes. Many corporate researchers are reluctant to consider viral vectors as a means of delivery due to the poor reputation of gene therapy, as a consequence of adverse events that occurred in early studies. More recently developed vector systems have excellent safety profiles as a result of systematic improvements in this key area. The time has come to re-consider viral vectors in order to overcome the complications and allow efficient, specific and well-tolerated delivery in RNAi applications.
Authors and Affiliations
Jens Kurreck
Keywords
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