CN112969790B - 用于递送核碱基编辑系统的组合物和方法 - Google Patents

用于递送核碱基编辑系统的组合物和方法 Download PDF

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CN112969790B
CN112969790B CN201980073055.6A CN201980073055A CN112969790B CN 112969790 B CN112969790 B CN 112969790B CN 201980073055 A CN201980073055 A CN 201980073055A CN 112969790 B CN112969790 B CN 112969790B
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ncas9
cas9
terminal fragment
domain
amino acid
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CN112969790A (zh
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D·布赖森
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Bim Medical Co ltd
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
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    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Enzymes And Modification Thereof (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201980073055.6A 2018-09-07 2019-09-07 用于递送核碱基编辑系统的组合物和方法 Active CN112969790B (zh)

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US201862728703P 2018-09-07 2018-09-07
US62/728,703 2018-09-07
US201862779404P 2018-12-13 2018-12-13
US62/779,404 2018-12-13
PCT/US2019/050111 WO2020051561A1 (en) 2018-09-07 2019-09-07 Compositions and methods for delivering a nucleobase editing system

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CN112969790B true CN112969790B (zh) 2024-07-05

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US (1) US12529041B2 (https=)
EP (1) EP3847254A4 (https=)
JP (2) JP7657711B2 (https=)
KR (1) KR20210055733A (https=)
CN (1) CN112969790B (https=)
AU (1) AU2019336245B2 (https=)
CA (1) CA3112011A1 (https=)
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