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1. WO2022088342 - SIRNA TARGETING FOXP3 GENE AND MODIFICATION METHOD THEREFOR

Publication Number WO/2022/088342
Publication Date 05.05.2022
International Application No. PCT/CN2020/131889
International Filing Date 26.11.2020
IPC
C12N 15/113 2010.1
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
A61K 31/713 2006.1
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
713Double-stranded nucleic acids or oligonucleotides
A61K 48/00 2006.1
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
A61P 1/00 2006.1
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
CPC
A61K 31/713
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
713Double-stranded nucleic acids or oligonucleotides
A61K 48/00
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
A61P 1/00
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
C12N 15/113
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; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
Applicants
  • 苏州吉玛基因股份有限公司 SUZHOU GENEPHARMA CO., LTD. [CN]/[CN]
Inventors
  • 胡荣宽 HU, Rongkuan
  • 张佩琢 ZHANG, Peizhuo
  • 张紫楠 ZHANG, Zinan
  • 李琴 LI, Qin
Agents
  • 北京三聚阳光知识产权代理有限公司 SUNSHINE INTELLECTUAL PROPERTY INTERNATIONAL CO., LTD.
Priority Data
202011174093.128.10.2020CN
Publication Language Chinese (zh)
Filing Language Chinese (ZH)
Designated States
Title
(EN) SIRNA TARGETING FOXP3 GENE AND MODIFICATION METHOD THEREFOR
(FR) ARNSI CIBLANT LE GÈNE FOXP3 ET SON PROCÉDÉ DE MODIFICATION
(ZH) 一种靶向FOXP3基因的siRNA及其修饰方法
Abstract
(EN) A double-stranded siRNA molecule is provided, the double-stranded siRNA molecule being A or B below: A, a double-stranded siRNA molecule formed via the complementing of an RNA single strand as shown by SEQ ID No. 6 (FOXP3-Homo-487: CACAUUUCAUGCACCAGCUTT) and an RNA single strand as shown by SEQ ID No. 7 (FOXP3-Homo-487: AGCUGGUGCAUGAAAUGUGTT); B, a double-stranded siRNA molecule formed via the complementing of the RNA single strand as shown by SEQ ID No. 7 and an RNA single strand having 60% or higher homology with the RNA single strand as shown by SEQ ID No. 6; or, a double-stranded siRNA molecule formed via the complementing of the RNA single strand as shown by SEQ ID No. 6 and an RNA single strand having 60% or higher homology with the RNA single strand as shown by SEQ ID No. 7; or, a double-stranded siRNA molecule formed via the complementing of an RNA single strand having 70% or higher homology with the RNA single strand as shown by SEQ ID No. 6 and an RNA single strand having 70% or higher homology with the RNA single strand as shown by SEQ ID No. 7.
(FR) L'invention concerne une molécule d'ARNsi double brin, la molécule d'ARNsi double brin étant A ou B ci-dessous : A, une molécule d'ARNsi double brin formée via la complémentation d'un ARN simple brin comme indiqué par SEQ ID NO : 6 (FOXP3-Homo-487 : CACAUUUCAUGCACCAGCUTT) et un ARN simple brin tel que représenté par SEQ ID NO : 7 (FOXP3-Homo-487 : AGCUGGUGCAUGAAAUGUGTT) ; B, une molécule d'ARNsi double brin formée par la complémentation de l'ARN simple brin tel que représenté par SEQ ID NO : 7 et un ARN simple brin ayant 60 % ou plus d'homologie avec l'ARN simple brin tel que représenté par SEQ ID NO : 6; ou, une molécule d'ARNsi double brin formée par la complémentation de l'ARN simple brin tel que représenté par SEQ ID NO : 6 et un ARN simple brin ayant 60 % ou plus d'homologie avec l'ARN simple brin tel que représenté par SEQ ID NO : 7; ou, une molécule d'ARNsi double brin formée par la complémentation d'un ARN simple brin ayant 70 % ou plus d'homologie avec l'ARN simple brin tel que représenté par SEQ ID NO : 6 et un ARN simple brin ayant 70 % ou plus d'homologie avec l'ARN simple brin tel que représenté par SEQ ID NO : 7.
(ZH) 提供一种双链siRNA分子,所述双链siRNA分子为如下A或B:A、SEQ ID No.6 (FOXP3-Homo-487:CACAUUUCAUGCACCAGCUTT) 所示的RNA单链和SEQ ID No.7 (FOXP3-Homo-487:AGCUGGUGCAUGAAAUGUGTT) 所示的RNA单链互补而成的双链siRNA分子;B、SEQ ID No.7所示的RNA单链和与SEQ ID No.6所示的RNA单链有60%以上同源性的RNA单链互补而成的双链siRNA分子;或,SEQ ID No.6所示的RNA单链和与SEQ ID No.7所示的RNA单链有60%以上同源性的RNA单链互补而成的双链siRNA分子;或,与SEQ ID No.6所示的RNA单链有70%以上同源性的RNA单链和与SEQ ID No.7所示的RNA单链有70%以上同源性的RNA单链互补而成的双链siRNA分子。
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