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Next‐gen mRNA‐enhanced Cell Therapy

John P Cooke,Anahita Mojiri,Rhonda Holgate,Brandon Walther, Hongye Li, Colleen Stubbs,Yi Liang, Celetta Callaway,Palas Chanda,Tulsi Damase, Claudia Kettlun-Leyton, Dan Kiss,Roman Sukhovershin

The FASEB Journal(2020)

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摘要
A new therapeutic field is emerging as obstacles are overcome to the clinical applications of mRNA drugs. The immunogenicity of mRNA has been reduced by the use of modified nucleosides and ultra‐purification. The stability and translatability of mRNA is increased by attention to optimizing codon use, the translational ramp, the 3′ and 5′UTRs, and by adding nuclease‐resistant secondary structure. The delivery of mRNA depends upon the application, but advances in targeted nanoparticle delivery vehicles, electro‐ or mechanoporation, microneedles and scaffolds have increased the delivery of therapeutic mRNA to the desired tissue. Because mRNA is non‐integrating, it is safer for human use than DNA‐based gene therapies. Cell therapies, including stem cell therapies for regenerative medicine, may be enhanced by mRNA‐based strategies. For example, in immunodeficient mice bearing a human B cell …
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关键词
siRNA Therapeutics,Therapeutic RNAi
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