KR20130018395A - 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 - Google Patents

헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 Download PDF

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KR20130018395A
KR20130018395A KR1020117027996A KR20117027996A KR20130018395A KR 20130018395 A KR20130018395 A KR 20130018395A KR 1020117027996 A KR1020117027996 A KR 1020117027996A KR 20117027996 A KR20117027996 A KR 20117027996A KR 20130018395 A KR20130018395 A KR 20130018395A
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oligonucleotide
antisense
modified
oligonucleotides
nucleic acid
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Korean (ko)
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조셉 콜라드
올가 크로코바 셔만
칼로스 코이토
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큐알엔에이, 인크.
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00—Antiparasitic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense

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KR1020117027996A 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료 Ceased KR20130018395A (ko)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US17471909P 2009-05-01 2009-05-01
PCT/US2010/033078 WO2010127195A2 (en) 2009-05-01 2010-04-30 Antisense oligonucleotides of hemoglobins

Related Child Applications (1)

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KR1020177011705A Division KR101857402B1 (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료

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KR1020117027996A Ceased KR20130018395A (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료
KR1020177011705A Expired - Fee Related KR101857402B1 (ko) 2009-05-01 2010-07-12 헤모글로빈(ｈｂｆ/ｈｂｇ)에 천연 안티센스 전사체의 억제에 의해 ｈｂｆ/ｈｂｇ 관련된 질환의 치료

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US (1) US8318690B2 (enExample)
EP (1) EP2424987B1 (enExample)
JP (1) JP2013524769A (enExample)
KR (2) KR20130018395A (enExample)
CN (1) CN102639151B (enExample)
CA (1) CA2760589C (enExample)
DK (1) DK2424987T3 (enExample)
ES (1) ES2661787T3 (enExample)
NO (1) NO2424987T3 (enExample)
WO (1) WO2010127195A2 (enExample)

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BR112014028644A2 (pt) 2012-05-16 2017-08-15 Rana Therapeutics Inc Composições e métodos para modulação da expressão de atp2a2
CA2873769A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
EP3511416A1 (en) 2012-05-16 2019-07-17 Translate Bio MA, Inc. Compositions and methods for modulating gene expression
BR112014028634A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
CN106456802B (zh) 2014-01-30 2019-10-18 儿童医院医疗中心 一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白
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US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US11179379B2 (en) * 2017-01-30 2021-11-23 The Children's Hospital Of Philadelphia Compositions and methods for hemoglobin production
EP4183882A1 (en) 2017-09-08 2023-05-24 MiNA Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
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