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Cell-Selective Gene Editing

The strategy reduces toxicity, has high flexibility and is able to edit multiple genes and target multiple cell types

Background

Modified mRNA-based therapeutics hold great promise for numerous untreatable diseases. The potential advantages of modified mRNA biomolecules include reduced toxicity, decreased activation of the innate immune pathway, and improved translation in virtually all cell types, including non-dividing cells by circumventing nuclear localiza-tion. Additionally, modified mRNA provides enormous flexibility with respect to production and application, because any protein with a known sequence can be encoded and expressed. Lastly, modified mRNA can be produced rapidly in a cell-free, cost-effective manner. While modified mRNA presents an intriguing therapeutic option for drug delivery and disease treatment, its potential silencing by small interfering RNA or microRNA remains an issue.

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