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1. WO2022000572 - METHOD FOR ACTIVATING EXPRESSION OF GAMMA-GLOBIN GENE, AND COMPOSITION

Publication Number WO/2022/000572
Publication Date 06.01.2022
International Application No. PCT/CN2020/101936
International Filing Date 14.07.2020
IPC
C12N 15/113 2010.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/85 2006.1
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
67General methods for enhancing the expression
C12N 15/12 2006.1
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C12N 5/10 2006.1
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10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
A61K 35/28 2015.1
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35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
CPC
A61K 35/28
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61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
A61P 7/06
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-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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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
67General methods for enhancing the expression
C12N 15/85
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
Applicants
  • 广州瑞风生物科技有限公司 GUANGZHOU REFORGENE MEDICINE CO. , LTD. [CN]/[CN]
Inventors
  • 梁峻彬 LIANG, Junbin
  • 古博 GU, Bo
  • 徐辉 XU, Hui
Agents
  • 广州三环专利商标代理有限公司 SCIHEAD IP LAW FIRM
Priority Data
202010616648.701.07.2020CN
Publication Language Chinese (zh)
Filing Language Chinese (ZH)
Designated States
Title
(EN) METHOD FOR ACTIVATING EXPRESSION OF GAMMA-GLOBIN GENE, AND COMPOSITION
(FR) PROCÉDÉ D'ACTIVATION DE L'EXPRESSION D'UN GÈNE DE GAMMA-GLOBINE ET COMPOSITION
(ZH) 激活γ-珠蛋白基因表达的方法和组合物
Abstract
(EN) Provided is a new method for activating transcription of a gamma-globin gene. The method uses a single-stranded oligonucleotide (ssODN) containing GATA or an antisense complementary sequence TATC thereof as guidance information, and performs gene editing in a gamma-globin gene regulatory region to form a GATA-containing enhancer element, which can promote the expression of the gamma-globin gene in mature red blood cells. Hematopoietic stem cells genetically edited by the method have normal functions, can significantly improve the expression of fetal hemoglobin after being differentiated into red blood cells, and therefore can be used in clinical treatment of beta-thalassemia and sickle cell anemia.
(FR) L'invention concerne un nouveau procédé servant à activer la transcription de gène de gamma-globine. Le procédé utilise un oligonucléotide simple brin (ssODN) contenant GATA ou une séquence complémentaire antisens TATC correspondante en tant qu'informations de guidage et réalise une édition de gène dans une région régulatrice de gène de gamma-globine pour former un élément activateur contenant GATA, qui peut favoriser l'expression du gène de la gamma-globine dans des globules rouges matures. Les cellules souches hématopoïétiques génétiquement éditées par le procédé ont des fonctions normales, peuvent améliorer significativement l'expression de l'hémoglobine fœtale après avoir été différenciées en globules rouges et peuvent donc être utilisées dans le traitement clinique de la bêta-thalassémie et de la drépanocytose.
(ZH) 提供了一种激活γ-珠蛋白基因转录的新方法,该方法采用含有GATA或其反义互补序列TATC的单链寡核苷酸(ssODN)作为指导信息,在γ-珠蛋白基因调控区进行基因编辑形成含有GATA的增强子元件,能够促进γ-珠蛋白基因在成熟红细胞中表达。利用该方法进行基因编辑后的造血干细胞具有正常功能,且在分化成红细胞后能显著提高胎儿血红蛋白的表达,因此可用于β-地中海贫血和镰刀红细胞贫血的临床治疗中。
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