CN105683376A - 用于治疗遗传病状的方法和组合物 - Google Patents

用于治疗遗传病状的方法和组合物 Download PDF

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Publication number
CN105683376A
CN105683376A CN201480039996.5A CN201480039996A CN105683376A CN 105683376 A CN105683376 A CN 105683376A CN 201480039996 A CN201480039996 A CN 201480039996A CN 105683376 A CN105683376 A CN 105683376A
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gene
cell
sequence
plant
cells
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Chinese (zh)
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A·康威
G·J·柯斯特
R·迪克勒弗
E·J·瑞巴
A·瑞克
F·诺弗
汪建斌
H·S·张
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Sangamo Therapeutics Inc
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Sangamo Biosciences Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)

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  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
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  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
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CN201480039996.5A 2013-05-15 2014-05-15 用于治疗遗传病状的方法和组合物 Pending CN105683376A (zh)

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US201361823689P 2013-05-15 2013-05-15
US61/823,689 2013-05-15
PCT/US2014/038216 WO2014186585A2 (en) 2013-05-15 2014-05-15 Methods and compositions for treatment of a genetic condition

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CN106244555A (zh) * 2016-08-23 2016-12-21 广州医科大学附属第三医院 一种提高基因打靶的效率的方法及β‑球蛋白基因位点的碱基原位修复方法
CN107267516A (zh) * 2017-07-28 2017-10-20 佛山科学技术学院 双sgRNA介导的基因精确修饰方法及应用
CN108504685A (zh) * 2018-03-27 2018-09-07 宜明细胞生物科技有限公司 一种利用CRISPR/Cas9系统同源重组修复IL-2RG缺陷基因的方法
CN108779462A (zh) * 2015-12-28 2018-11-09 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法
CN110114077A (zh) * 2016-09-13 2019-08-09 中央研究院 藉移植增加寿命及/或治疗细胞增殖疾病的方法
CN110536963A (zh) * 2017-01-26 2019-12-03 桑格摩生物治疗股份有限公司 B细胞工程改造
CN111108207A (zh) * 2017-06-02 2020-05-05 国家健康与医学研究院 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法
CN111565730A (zh) * 2017-11-09 2020-08-21 桑格摩生物治疗股份有限公司 细胞因子诱导型含sh2蛋白（cish）基因的遗传修饰
CN113544270A (zh) * 2018-12-23 2021-10-22 美国杰特贝林生物制品有限公司 用于递送细胞调节剂的纳米胶囊
CN113646006A (zh) * 2019-01-18 2021-11-12 国立大学法人大阪大学 用于营养不良型大疱性表皮松解症的治疗剂
CN113924102A (zh) * 2019-06-18 2022-01-11 Tx医生公司 同种异体car-t细胞疗法
CN114521214A (zh) * 2020-07-24 2022-05-20 尔知渃米斯股份有限公司 视紫红质转录体特异性反式剪接核酶及其用途
CN116179493A (zh) * 2021-09-29 2023-05-30 成都美杰赛尔生物科技有限公司 敲除两种免疫检测点基因的免疫细胞及其制备方法与应用

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