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Synthetic RNA for use in the treatment of dystrophic epidermolysis bullosa
Synthetic RNA for use in the treatment of dystrophic epidermolysis bullosa
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机译:用于治疗营养不良性大疱性表皮松解的合成RNA
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摘要
The present invention relates in part to nucleic acids, including nucleic acids encoding proteins, therapeutics and cosmetics comprising nucleic acids, methods for delivering nucleic acids to cells, tissues, organs, and patients, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, therapeutics, and cosmetics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules, including cells presentin vivo.Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
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