MA50844A - Méthode pour augmenter le niveau d'expression d'hémoglobine foetale - Google Patents

Méthode pour augmenter le niveau d'expression d'hémoglobine foetale

Info

Publication number
MA50844A
MA50844A MA050844A MA50844A MA50844A MA 50844 A MA50844 A MA 50844A MA 050844 A MA050844 A MA 050844A MA 50844 A MA50844 A MA 50844A MA 50844 A MA50844 A MA 50844A
Authority
MA
Morocco
Prior art keywords
increasing
level
fetal hemoglobin
hemoglobin expression
expression
Prior art date
Application number
MA050844A
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 MA50844A publication Critical patent/MA50844A/fr

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    • C12N5/0602—Vertebrate cells
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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
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    • 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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    • 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
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
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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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MA050844A 2017-10-27 2018-10-26 Méthode pour augmenter le niveau d'expression d'hémoglobine foetale MA50844A (fr)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
MA50844A true MA50844A (fr) 2020-09-02

Family

ID=66246216

Family Applications (2)

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
MA052665A MA52665A (fr) 2017-10-27 2018-10-26 Procédé d'amélioration de l'expression de l'hémoglobine foetale

Family Applications After (1)

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

Country Status (9)

Country Link
US (2) US20200384032A1 (fr)
EP (2) EP3702459B1 (fr)
JP (4) JP2021502062A (fr)
CN (10) CN109722414A (fr)
MA (2) MA50844A (fr)
PH (2) PH12020550488A1 (fr)
TW (2) TW201922777A (fr)
WO (2) WO2019080917A1 (fr)
ZA (2) ZA202002203B (fr)

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MX2018007987A (es) * 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.
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
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 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
CN114222816A (zh) * 2019-08-29 2022-03-22 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
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
AU2020342207A1 (en) * 2019-09-04 2022-04-21 Edigene (Guangzhou) Inc. Method for evaluating gene editing therapy based on off-target assessment
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