MA52665A - Procédé d'amélioration de l'expression de l'hémoglobine foetale - Google Patents

Procédé d'amélioration de l'expression de l'hémoglobine foetale

Info

Publication number
MA52665A
MA52665A MA052665A MA52665A MA52665A MA 52665 A MA52665 A MA 52665A MA 052665 A MA052665 A MA 052665A MA 52665 A MA52665 A MA 52665A MA 52665 A MA52665 A MA 52665A
Authority
MA
Morocco
Prior art keywords
expression
improving
fetal hemoglobin
hemoglobin
fetal
Prior art date
Application number
MA052665A
Other languages
English (en)
Inventor
Riguo Fang
Fucai Liang
Jia Wang
Penghui Xia
Lingling Yu
Pengfei Yuan
Original Assignee
Edigene Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Edigene Inc filed Critical Edigene Inc
Publication of MA52665A publication Critical patent/MA52665A/fr

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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/18—Erythrocytes
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • 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
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
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    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA052665A 2017-10-27 2018-10-26 Procédé d'amélioration de l'expression de l'hémoglobine foetale MA52665A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN201711027708 2017-10-27

Publications (1)

Publication Number Publication Date
MA52665A true MA52665A (fr) 2020-09-02

Family

ID=66246216

Family Applications (2)

Application Number Title Priority Date Filing Date
MA052665A MA52665A (fr) 2017-10-27 2018-10-26 Procédé d'amélioration de l'expression de l'hémoglobine foetale
MA050844A MA50844A (fr) 2017-10-27 2018-10-26 Méthode pour augmenter le niveau d'expression d'hémoglobine foetale

Family Applications After (1)

Application Number Title Priority Date Filing Date
MA050844A MA50844A (fr) 2017-10-27 2018-10-26 Méthode pour augmenter le niveau d'expression d'hémoglobine foetale

Country Status (9)

Country Link
US (2) US20200339979A1 (fr)
EP (2) EP3702458A4 (fr)
JP (4) JP2021500058A (fr)
CN (10) CN109722414A (fr)
MA (2) MA52665A (fr)
PH (2) PH12020550488A1 (fr)
TW (2) TW201930592A (fr)
WO (2) WO2019080917A1 (fr)
ZA (2) ZA202002203B (fr)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
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SG11201805217XA (en) * 2015-12-28 2018-07-30 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
CN109486814A (zh) * 2017-10-31 2019-03-19 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
EP3788144B1 (fr) 2018-05-01 2025-09-24 The Children's Medical Center Corporation Administration et édition améliorées de bcl11a rnp/crispr à l'aide d'une 3xnls-cas9
CN112011576A (zh) * 2019-05-31 2020-12-01 华东师范大学 Crispr基因编辑技术在治疗地中海贫血中的应用
CN112391410B (zh) * 2020-02-17 2024-04-02 华东师范大学 一种sgRNA及其在修复内含子异常剪接中的应用
CN110511909B (zh) * 2019-07-29 2022-01-04 吉林大学 体外扩增造血干细胞的生长因子组合物及其应用
CN114364799B (zh) * 2019-08-28 2024-07-19 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
WO2021037234A1 (fr) * 2019-08-29 2021-03-04 广州辑因医疗科技有限公司 Procédé pour réparer efficacement une mutation génétique responsable d'une anémie avec sidéroblastes en couronne
CN112442516A (zh) * 2019-08-29 2021-03-05 广州辑因医疗科技有限公司 一种高效修复环状铁粒幼细胞性贫血基因突变的方法
KR20220053671A (ko) * 2019-09-04 2022-04-29 에디진 인크. 오프 타겟 평가 기반의 유전자 편집 치료의 평가 방법
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