DK3145553T3 - Lille interfererende rna (sirna) til behandling af type 2 (ado2) autosomal dominant osteopetrose forårsaget af clcn7 (ado2 clcn7-afhængig)-genmutation - Google Patents

Lille interfererende rna (sirna) til behandling af type 2 (ado2) autosomal dominant osteopetrose forårsaget af clcn7 (ado2 clcn7-afhængig)-genmutation Download PDF

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Publication number
DK3145553T3
DK3145553T3 DK15732387T DK15732387T DK3145553T3 DK 3145553 T3 DK3145553 T3 DK 3145553T3 DK 15732387 T DK15732387 T DK 15732387T DK 15732387 T DK15732387 T DK 15732387T DK 3145553 T3 DK3145553 T3 DK 3145553T3
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Prior art keywords
ado2
clcn7
osteopetrose
genutation
sirna
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DK15732387T
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English (en)
Inventor
Mattia Capulli
Antonio Maurizi
Nadia Rucci
Anna Maria Teti
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Univ Degli Studi Dellaquila
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Publication of DK3145553T3 publication Critical patent/DK3145553T3/da

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    • C—CHEMISTRY; METALLURGY
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (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)
DK15732387T 2014-05-23 2015-05-21 Lille interfererende rna (sirna) til behandling af type 2 (ado2) autosomal dominant osteopetrose forårsaget af clcn7 (ado2 clcn7-afhængig)-genmutation DK3145553T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ITRM20140272 2014-05-23
PCT/IB2015/053730 WO2015177743A1 (en) 2014-05-23 2015-05-21 Small interfering rna (sirna) for the therapy of type 2 (ado2) autosomal dominant osteopetrosis caused by clcn7 (ado2 clcn7-dependent) gene mutation

Publications (1)

Publication Number Publication Date
DK3145553T3 true DK3145553T3 (da) 2019-10-28

Family

ID=51220775

Family Applications (1)

Application Number Title Priority Date Filing Date
DK15732387T DK3145553T3 (da) 2014-05-23 2015-05-21 Lille interfererende rna (sirna) til behandling af type 2 (ado2) autosomal dominant osteopetrose forårsaget af clcn7 (ado2 clcn7-afhængig)-genmutation

Country Status (11)

Country Link
US (1) US10450571B2 (da)
EP (1) EP3145553B1 (da)
JP (1) JP6795492B2 (da)
AU (1) AU2015262889B2 (da)
CA (1) CA2949345C (da)
DK (1) DK3145553T3 (da)
ES (1) ES2753355T3 (da)
HU (1) HUE046546T2 (da)
PL (1) PL3145553T3 (da)
PT (1) PT3145553T (da)
WO (1) WO2015177743A1 (da)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017004526A1 (en) * 2015-07-02 2017-01-05 University Of Louisville Research Foundation, Inc. EDIBLE PLANT-DERIVED MICROVESICLE COMPOSITIONS FOR DELIVERY OF miRNA AND METHODS FOR TREATMENT OF CANCER
CN111235152A (zh) * 2020-03-31 2020-06-05 深圳市人民医院 特异性靶向CLCN7的sgRNA及其应用

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002079414A2 (en) * 2001-03-29 2002-10-10 Deltagen, Inc. Transgenic mice containing clcn7 chloride channel gene disruptions
WO2003062821A1 (en) * 2002-01-23 2003-07-31 Bristol-Myers Squibb Company Modulators of the clc-7 chloride channel and methods for their identification and use in the treatment and prevention of osteoporosis and related disease states
DK2284266T3 (da) * 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53

Also Published As

Publication number Publication date
CA2949345A1 (en) 2015-11-26
US20170101644A1 (en) 2017-04-13
EP3145553A1 (en) 2017-03-29
WO2015177743A1 (en) 2015-11-26
PL3145553T3 (pl) 2020-03-31
PT3145553T (pt) 2019-10-30
US10450571B2 (en) 2019-10-22
AU2015262889A8 (en) 2017-02-02
HUE046546T2 (hu) 2020-03-30
AU2015262889B2 (en) 2020-04-23
ES2753355T3 (es) 2020-04-08
EP3145553B1 (en) 2019-07-31
AU2015262889A1 (en) 2016-12-01
CA2949345C (en) 2022-06-14
JP6795492B2 (ja) 2020-12-02
JP2017521094A (ja) 2017-08-03

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