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DELIVERY, USE AND USE IN THERAPY OF CRISPR-CAS SYSTEMS AND COMPOSITIONS FOR TARGETED ACTION ON DISORDERS AND DISEASES USING VIRAL COMPONENTS

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国家林业和草原科学数据中心2023-02-12 更新2024-03-07 收录
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FIELD: biotechnology. SUBSTANCE: disclosed is a composition containing one or more viral vectors coding CRISPR/Cas system for use in treating a disease or cerebral disorder. EFFECT: said system due to the presence of at least one nuclear localization signal (NLS) enables efficient editing of the eukaryotic cell genome, in connection with which it can be used to edit genes associated with cerebral diseases in mammals. 20 cl, 85 dwg, 18 tbl, 40 ex

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2023-02-12
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