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1. WO2020063178 - GENE EDITING COMPOSITION OR KIT USED FOR IN VIVO GENE THERAPY

Publication Number WO/2020/063178
Publication Date 02.04.2020
International Application No. PCT/CN2019/100809
International Filing Date 15.08.2019
IPC
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 9/22 2006.01
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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
A61P 9/10 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
10for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
A61P 21/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
CPC
A61K 31/7088
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
A61P 1/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
1Drugs for disorders of the alimentary tract or the digestive system
16for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
A61P 13/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
12of the kidneys
A61P 21/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
A61P 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 9/10
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
10for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
Applicants
  • 中国科学技术大学 UNIVERSITY OF SCIENCE AND TECHNOLOGY OF CHINA [CN]/[CN]
Inventors
  • 薛天 XUE, Tian
Agents
  • 中科专利商标代理有限责任公司 CHINA SCIENCE PATENT & TRADEMARK AGENT LTD.
Priority Data
201811144025.326.09.2018CN
Publication Language Chinese (ZH)
Filing Language Chinese (ZH)
Designated States
Title
(EN) GENE EDITING COMPOSITION OR KIT USED FOR IN VIVO GENE THERAPY
(FR) COMPOSITION OU KIT D'ÉDITION DE GÈNES UTILISÉ POUR UNE THÉRAPIE GÉNIQUE IN VIVO
(ZH) 用于在体基因治疗的基因编辑组合物或试剂盒
Abstract
(EN)
A gene editing composition or kit used for in vivo gene therapy, comprising: 1) sgRNA that is linked to a nucleic acid molecule of a binding protein and that targets a target mutant gene, or a coding sequence thereof, 2) a template nucleic acid that repairs the target mutant gene, or a coding sequence thereof, 3) an sgRNA-guided nuclease, or a coding sequence thereof, 4) a homologous recombination-promoting protein that is fused with the nucleic acid binding protein, or a coding sequence thereof, wherein the nucleic acid binding protein is capable of binding to the nucleic acid molecule in 1) above. The sgRNA, composition or kit may be used to treat diseases caused by genetic mutations, such as hereditary diseases. The sgRNA, composition or kit may effectively improve the efficiency of homologous recombination repair, and may also achieve homologous recombination repair in non-dividing cells.
(FR)
L'invention concerne une composition ou un kit d'édition de gènes utilisé pour une thérapie génique in vivo, comprenant : 1) un ARNsg lié à une molécule d'acide nucléique d'une protéine de liaison et ciblant un gène mutant cible ou une séquence de codage de celui-ci, 2) un acide nucléique de matrice qui répare le gène mutant cible ou une séquence de codage de celui-ci, 3) une nucléase guidée par l'ARNsg ou une séquence de codage de celle-ci, 4) une protéine homologue favorisant la recombinaison qui est fusionnée avec la protéine de liaison à l'acide nucléique ou une séquence de codage de celle-ci, la protéine de liaison à l'acide nucléique étant susceptible de se lier à la molécule d'acide nucléique décrite en 1) ci-dessus. L'ARNsg, la composition ou le kit peuvent être utilisés pour traiter des maladies provoquées par des mutations génétiques, telles que des maladies héréditaires. L'ARNsg, la composition ou le kit peuvent améliorer efficacement l'efficacité de la réparation de recombinaison homologue et peuvent également obtenir une réparation de recombinaison homologue dans des cellules ne divisant pas.
(ZH)
一种用于在体基因治疗的基因编辑组合物或试剂盒,其包含1)靶向目的突变基因的与结合蛋白的核酸分子连接的sgRNA或其编码序列,2)修复目的突变基因的模板核酸或其编码序列,3)sgRNA引导的核酸酶或其编码序列,4)与核酸结合蛋白融合的促进同源重组的蛋白或其编码序列,其中所述核酸结合蛋白能够与上述1)中的核酸分子结合。所述sgRNA、组合物或试剂盒可用于治疗基因突变导致的疾病如遗传性疾病。所述sgRNA、组合物或试剂盒能够有效的提高同源重组修复的效率,并且在非分裂细胞内也能实现同源重组修复。
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