HUE046546T2 - Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére - Google Patents

Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére

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Publication number
HUE046546T2
HUE046546T2 HUE15732387A HUE15732387A HUE046546T2 HU E046546 T2 HUE046546 T2 HU E046546T2 HU E15732387 A HUE15732387 A HU E15732387A HU E15732387 A HUE15732387 A HU E15732387A HU E046546 T2 HUE046546 T2 HU E046546T2
Authority
HU
Hungary
Prior art keywords
ado2
clcn7
sirna
therapy
dependent
Prior art date
Application number
HUE15732387A
Other languages
English (en)
Inventor
Anna Maria Teti
Nadia Rucci
Mattia Capulli
Antonio Maurizi
Original Assignee
Univ Degli Studi Dellaquila
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Degli Studi Dellaquila filed Critical Univ Degli Studi Dellaquila
Publication of HUE046546T2 publication Critical patent/HUE046546T2/hu

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
  • 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)
HUE15732387A 2014-05-23 2015-05-21 Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére HUE046546T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
ITRM20140272 2014-05-23

Publications (1)

Publication Number Publication Date
HUE046546T2 true HUE046546T2 (hu) 2020-03-30

Family

ID=51220775

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE15732387A HUE046546T2 (hu) 2014-05-23 2015-05-21 Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére

Country Status (11)

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

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
DK3145553T3 (da) 2019-10-28
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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HUE046546T2 (hu) Kis interferáló RNS (siRNS) CLCN7 (ADO2 CLCN7-függõ) génmutáció által okozott 2-es típusú (ADO2) autoszómás domináns oszteopetrózis kezelésére