Therapeutic Delivery of mRNA: The Medium Is the Message

Messenger RNA (mRNA) has several advantages over DNA for gene transfer and expression, including the lack of any requirement for nuclear localization or transcription and the nearly negligible possibility of genomic integration of the delivered sequence. However, the labile nature of mRNA and its capacity to elicit innate immune responses are important limitations to its in vivo application. Now, an article by Kormann et al.1 published in Nature Biotechnology addresses these drawbacks and provides new hope for the potential use of mRNA as a source of therapeutic gene product in vivo.