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Safe, effective and targeting viral vector is the key factor for successful clinical gene therapy. microRNA, a class of small, single-stranded endogenous RNAs, act as post-transcriptional regulators of gene expression.The discovery of this kind regulatory elements provide a new field for targeting regulation in gene therapy. Here, we elucidated the principle of microRNA in regulation of targeting viral vector. Then, the applications of microRNA in the fields of elimination contamination from replication competent virus, reduction of transgene-specific immunity, promotion of cancer-targeted gene therapy and development of live attenuated vaccines were further discussed.

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Safe, effective and targeting viral vector is the key to successful application of clinical gene therapy. The discovery of microRNA has blazed a trail for targeting regulation in gene therapy. microRNA is a class of small, single-stranded endogenous RNAs and act as post-transcriptional regulators of gene expression. At the present study, the emphases are placed on the applications of microRNA in the fields of elimination contamination from replication competent virus, reduction of transgene-specific immunity, promotion of cancer-targeted gene therapy and development of live attenuated vaccines based on the principle of microRNA in regulation of targeting viral vector.
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