CN104284669A - 治疗血红蛋白病的组合物和方法 - Google Patents

治疗血红蛋白病的组合物和方法 Download PDF

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Publication number
CN104284669A
CN104284669A CN201380017193.5A CN201380017193A CN104284669A CN 104284669 A CN104284669 A CN 104284669A CN 201380017193 A CN201380017193 A CN 201380017193A CN 104284669 A CN104284669 A CN 104284669A
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seq
endonuclease
homing endonuclease
amino acid
region
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迈克尔·A·本德尔
马克·T·格劳迪尼
巴里·L·斯托达德
亮武内
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Fred Hutchinson Cancer Center
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201380017193.5A 2012-02-24 2013-02-22 治疗血红蛋白病的组合物和方法 Pending CN104284669A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261603231P 2012-02-24 2012-02-24
US61/603,231 2012-02-24
PCT/US2013/027459 WO2013126794A1 (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

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CN104284669A true CN104284669A (zh) 2015-01-14

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US (2) US20150166969A1 (https=)
EP (2) EP2836226B1 (https=)
JP (2) JP6170080B2 (https=)
KR (2) KR20180009383A (https=)
CN (1) CN104284669A (https=)
AU (2) AU2013222170B2 (https=)
BR (1) BR112014020625A2 (https=)
CA (1) CA2865129A1 (https=)
DK (1) DK2836226T3 (https=)
ES (1) ES2641840T3 (https=)
HK (1) HK1249861A1 (https=)
IL (2) IL234225B (https=)
IN (1) IN2014DN07853A (https=)
NZ (1) NZ630900A (https=)
RU (2) RU2650811C2 (https=)
SG (2) SG10201606959PA (https=)
WO (1) WO2013126794A1 (https=)
ZA (1) ZA201406187B (https=)

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CN104805118A (zh) * 2015-04-22 2015-07-29 扬州大学 一种苏禽黄鸡胚胎干细胞特定基因进行靶向敲除方法
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
CN107532168A (zh) * 2015-02-23 2018-01-02 克里斯珀医疗股份公司 治疗血红蛋白病的材料和方法
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
CN109735497A (zh) * 2017-10-27 2019-05-10 博雅辑因（北京）生物科技有限公司 一种提高胎儿血红蛋白表达水平的方法
CN109923211A (zh) * 2016-09-08 2019-06-21 蓝鸟生物公司 Pd-1归巢核酸内切酶变体、组合物及使用方法
CN110042124A (zh) * 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
CN110546262A (zh) * 2017-02-06 2019-12-06 诺华股份有限公司 用于治疗血红蛋白病的组合物和方法
CN111629747A (zh) * 2017-12-05 2020-09-04 沃泰克斯药物股份有限公司 Crispr-cas9修饰的cd34+人血色素干细胞和祖细胞及其用途
CN112469823A (zh) * 2018-04-27 2021-03-09 西雅图儿童医院d/b/a西雅图儿童研究所 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑
CN112979823A (zh) * 2019-12-18 2021-06-18 华东师范大学 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
CN114072518A (zh) * 2019-06-28 2022-02-18 Asc治疗公司 用于治疗地中海贫血或镰状细胞病的方法和组合物
CN114096666A (zh) * 2019-02-13 2022-02-25 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
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