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filingDate 2020-12-24-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2021-05-14-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber CN-112795595-A
titleOfInvention A gene therapy system for hereditary transthyretin amyloidosis
abstract The invention provides a gene therapy system for hereditary transthyretin amyloidosis disease. The CRISPR/Cas9 system is used to treat hATTR, and Nme2Cas9 which can be applied to human transthyretin (TTR) gene therapy for the disease is selected. Applied to this system, high-efficiency gRNA was optimized and designed to knock out functional genes, showing a strong ability to inhibit the expression of mutant TTR, showing the application of this system in the treatment of hereditary transthyretin amyloidosis diseases prospect.
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