AU2013287353B2 - Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell - Google Patents

Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell Download PDF

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Publication number
AU2013287353B2
AU2013287353B2 AU2013287353A AU2013287353A AU2013287353B2 AU 2013287353 B2 AU2013287353 B2 AU 2013287353B2 AU 2013287353 A AU2013287353 A AU 2013287353A AU 2013287353 A AU2013287353 A AU 2013287353A AU 2013287353 B2 AU2013287353 B2 AU 2013287353B2
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rna
seq
artificial
oligonucleotide
cftr
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AU2013287353A1 (en
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Daniel Anton DE BOER
Tita RITSEMA
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ProQR Therapeutics II BV
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2013287353A 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell Ceased AU2013287353B2 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261670681P 2012-07-12 2012-07-12
US61/670,681 2012-07-12
US201261718801P 2012-10-26 2012-10-26
US61/718,801 2012-10-26
EP13166465.8 2013-05-03
EP13166465 2013-05-03
EP13172515 2013-06-18
EP13172515.2 2013-06-18
EP13173818 2013-06-26
EP13173818.9 2013-06-26
PCT/NL2013/050534 WO2014011053A1 (en) 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell

Publications (2)

Publication Number Publication Date
AU2013287353A1 AU2013287353A1 (en) 2015-02-26
AU2013287353B2 true AU2013287353B2 (en) 2018-11-08

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AU2013287353A Ceased AU2013287353B2 (en) 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell

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US (2) US9605255B2 (https=)
EP (2) EP2852668B1 (https=)
JP (1) JP6373833B2 (https=)
KR (1) KR20150037968A (https=)
CN (1) CN104640986B (https=)
AU (1) AU2013287353B2 (https=)
BR (1) BR112015000710A8 (https=)
CA (1) CA2878934A1 (https=)
CY (1) CY1117858T1 (https=)
DK (2) DK2852668T3 (https=)
ES (2) ES2584856T3 (https=)
HR (1) HRP20160854T1 (https=)
HU (1) HUE029977T2 (https=)
IL (1) IL236654B (https=)
MX (1) MX356625B (https=)
NZ (1) NZ703824A (https=)
PL (1) PL2852668T3 (https=)
PT (1) PT2852668T (https=)
RS (1) RS54944B1 (https=)
RU (1) RU2663110C2 (https=)
SI (1) SI2852668T1 (https=)
SM (1) SMT201600234B (https=)
WO (1) WO2014011053A1 (https=)

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EP2852668B1 (en) 2012-07-12 2016-04-27 ProQR Therapeutics II B.V. Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell
RU2744460C2 (ru) 2014-04-15 2021-03-09 Вертекс Фармасьютикалз Инкорпорейтед Фармацевтические композиции для лечения заболеваний, опосредованных муковисцидозным трансмембранным регулятором проводимости
KR102589295B1 (ko) 2014-05-14 2023-10-13 타르그이뮨 테라퓨틱스 아게 개선된 폴리에틸렌이민 폴리에틸렌글리콜 벡터
CN107250113B (zh) 2014-10-07 2019-03-29 弗特克斯药品有限公司 囊性纤维化跨膜传导调节蛋白的调节剂的共晶
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US10676737B2 (en) * 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
US20210401869A1 (en) * 2015-02-20 2021-12-30 Rosalind Franklin University Of Medicine And Science Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
EP3359667A1 (en) 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
US10801026B2 (en) 2015-10-15 2020-10-13 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
JP6872560B2 (ja) * 2016-05-06 2021-05-19 エム． ウルフ、トッド プログラム可能ヌクレアーゼのあるゲノム編集及びプログラム可能ヌクレアーゼのないゲノム編集のための改善された方法
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
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CN107267516B (zh) * 2017-07-28 2020-09-29 佛山科学技术学院 双sgRNA介导的基因精确修饰方法及应用
WO2019076919A1 (en) * 2017-10-17 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) COMBINED TREATMENT OF CYSTIC FIBROSIS
EP3615085A4 (en) * 2018-02-09 2021-01-13 Ohio State Innovation Foundation RNA NANOSTRUCTURES, METHOD OF MANUFACTURING AND USES THEREOF
US11866702B2 (en) 2018-03-27 2024-01-09 University Of Rochester Nucleic acid molecules for pseudouridylation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
KR20210129646A (ko) 2019-01-22 2021-10-28 코로 바이오, 인크. Rna-편집 올리고뉴클레오타이드 및 그의 용도
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CN113397996A (zh) * 2021-07-23 2021-09-17 河南省人民医院 一种抗菌漱口水及其制备方法
CN114917227B (zh) * 2022-05-19 2023-10-31 中国人民解放军海军军医大学 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用
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HUE029977T2 (en) 2017-04-28
IL236654A0 (en) 2015-02-26
BR112015000710A8 (pt) 2018-04-03
US9994856B2 (en) 2018-06-12
MX2015000506A (es) 2015-06-03
RU2015104763A (ru) 2016-08-27
PL2852668T3 (pl) 2016-11-30
KR20150037968A (ko) 2015-04-08
SMT201600234B (it) 2016-08-31
CY1117858T1 (el) 2017-05-17
CN104640986B (zh) 2018-08-28
US9605255B2 (en) 2017-03-28
JP2015523082A (ja) 2015-08-13
DK2852668T3 (en) 2016-05-30
SI2852668T1 (sl) 2016-09-30
CA2878934A1 (en) 2014-01-16
AU2013287353A1 (en) 2015-02-26
EP2852668A1 (en) 2015-04-01
PT2852668T (pt) 2016-07-13
EP3103872B1 (en) 2018-08-29
ES2584856T3 (es) 2016-09-29
NZ703824A (en) 2018-06-29
US20170152518A1 (en) 2017-06-01
HRP20160854T1 (hr) 2016-09-23
IL236654B (en) 2018-02-28
RS54944B1 (sr) 2016-11-30
ES2698522T3 (es) 2019-02-05
JP6373833B2 (ja) 2018-08-15
EP3103872A1 (en) 2016-12-14
MX356625B (es) 2018-06-06
DK3103872T3 (en) 2018-10-22
CN104640986A (zh) 2015-05-20
BR112015000710A2 (https=) 2017-08-15
WO2014011053A1 (en) 2014-01-16
RU2663110C2 (ru) 2018-08-01
US20150197744A1 (en) 2015-07-16
EP2852668B1 (en) 2016-04-27
HK1208491A1 (zh) 2016-03-04

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