PL2852668T3 - Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce - Google Patents

Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce

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Publication number
PL2852668T3
PL2852668T3 PL13760133.2T PL13760133T PL2852668T3 PL 2852668 T3 PL2852668 T3 PL 2852668T3 PL 13760133 T PL13760133 T PL 13760133T PL 2852668 T3 PL2852668 T3 PL 2852668T3
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PL
Poland
Prior art keywords
oligonucleotides
sequence
making
change
rna molecule
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PL13760133.2T
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English (en)
Inventor
Boer Daniel Anton De
Tita Ritsema
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Publication of PL2852668T3 publication Critical patent/PL2852668T3/pl

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/08Solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
PL13760133.2T 2012-07-12 2013-07-12 Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce PL2852668T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261670681P 2012-07-12 2012-07-12
US201261718801P 2012-10-26 2012-10-26
EP13166465 2013-05-03
EP13172515 2013-06-18
EP13173818 2013-06-26

Publications (1)

Publication Number Publication Date
PL2852668T3 true PL2852668T3 (pl) 2016-11-30

Family

ID=49916369

Family Applications (1)

Application Number Title Priority Date Filing Date
PL13760133.2T PL2852668T3 (pl) 2012-07-12 2013-07-12 Oligonukleotydy do wprowadzania zmian w sekwencji docelowej cząsteczki RNA obecnej w żywej komórce

Country Status (23)

Country Link
US (2) US9605255B2 (pl)
EP (2) EP2852668B1 (pl)
JP (1) JP6373833B2 (pl)
KR (1) KR20150037968A (pl)
CN (1) CN104640986B (pl)
AU (1) AU2013287353B2 (pl)
BR (1) BR112015000710A8 (pl)
CA (1) CA2878934A1 (pl)
CY (1) CY1117858T1 (pl)
DK (2) DK2852668T3 (pl)
ES (2) ES2698522T3 (pl)
HR (1) HRP20160854T1 (pl)
HU (1) HUE029977T2 (pl)
IL (1) IL236654B (pl)
MX (1) MX356625B (pl)
NZ (1) NZ703824A (pl)
PL (1) PL2852668T3 (pl)
PT (1) PT2852668T (pl)
RS (1) RS54944B1 (pl)
RU (1) RU2663110C2 (pl)
SI (1) SI2852668T1 (pl)
SM (1) SMT201600234B (pl)
WO (1) WO2014011053A1 (pl)

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CA2944140C (en) 2014-04-15 2022-10-04 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases
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GB201418892D0 (en) * 2014-10-23 2014-12-10 Proqr Therapeutics B V DNA editing
EP3230445B1 (en) * 2014-12-12 2024-01-24 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
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US20210401869A1 (en) * 2015-02-20 2021-12-30 Rosalind Franklin University Of Medicine And Science Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
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CN108367021A (zh) * 2015-10-15 2018-08-03 希望之城 包含硫代磷酸化寡脱氧核苷酸的化合物和组合物及其使用方法
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
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PL3507366T3 (pl) * 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
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
US11261445B2 (en) 2017-10-17 2022-03-01 Inserm (Institut National De La Sante Et De La Recherche Medicale) Combination treatment for cystic fibrosis
US11976092B2 (en) * 2018-02-09 2024-05-07 Ohio State Innovation Foundation RNA nanostructures, methods of making, and uses thereof
JP7478923B2 (ja) 2018-03-27 2024-05-08 ユニバーシティ オブ ロチェスター シュードウリジン化のための核酸分子
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
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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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Also Published As

Publication number Publication date
RS54944B1 (sr) 2016-11-30
WO2014011053A1 (en) 2014-01-16
BR112015000710A2 (pl) 2017-08-15
DK3103872T3 (en) 2018-10-22
US20170152518A1 (en) 2017-06-01
CY1117858T1 (el) 2017-05-17
MX2015000506A (es) 2015-06-03
CA2878934A1 (en) 2014-01-16
CN104640986A (zh) 2015-05-20
JP2015523082A (ja) 2015-08-13
EP2852668A1 (en) 2015-04-01
NZ703824A (en) 2018-06-29
EP2852668B1 (en) 2016-04-27
AU2013287353A1 (en) 2015-02-26
CN104640986B (zh) 2018-08-28
RU2663110C2 (ru) 2018-08-01
HRP20160854T1 (hr) 2016-09-23
ES2584856T3 (es) 2016-09-29
IL236654B (en) 2018-02-28
EP3103872B1 (en) 2018-08-29
EP3103872A1 (en) 2016-12-14
SMT201600234B (it) 2016-08-31
BR112015000710A8 (pt) 2018-04-03
US9605255B2 (en) 2017-03-28
RU2015104763A (ru) 2016-08-27
HUE029977T2 (en) 2017-04-28
US9994856B2 (en) 2018-06-12
DK2852668T3 (en) 2016-05-30
HK1208491A1 (zh) 2016-03-04
SI2852668T1 (sl) 2016-09-30
MX356625B (es) 2018-06-06
US20150197744A1 (en) 2015-07-16
KR20150037968A (ko) 2015-04-08
ES2698522T3 (es) 2019-02-05
JP6373833B2 (ja) 2018-08-15
AU2013287353B2 (en) 2018-11-08
IL236654A0 (en) 2015-02-26
PT2852668T (pt) 2016-07-13

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