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

Info

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
Authority
DK
Denmark
Prior art keywords
ado2
clcn7
genutation
osteopetrose
sirna
Prior art date
Application number
DK15732387T
Other languages
English (en)
Inventor
Mattia Capulli
Antonio Maurizi
Nadia Rucci
Anna Maria Teti
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
Priority to ITRM20140272 priority Critical
Application filed by Univ Degli Studi Dellaquila filed Critical Univ Degli Studi Dellaquila
Priority to PCT/IB2015/053730 priority patent/WO2015177743A1/en
Application granted granted Critical
Publication of DK3145553T3 publication Critical patent/DK3145553T3/da

Links

Classifications

    • 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
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)

Priority Applications (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) CA2949345A1 (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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2002306942A1 (en) * 2001-03-29 2002-10-15 Deltagen, Inc. Transgenic mice containing clcn7 chloride channel gene disruptions
US20030215787A1 (en) 2002-01-23 2003-11-20 Wen-Pin Yang 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
PT2284266E (pt) * 2002-11-14 2013-12-17 Thermo Fisher Scient Biosciences Inc Siarn contra tp53

Also Published As

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

Similar Documents

Publication Publication Date Title
MA40587A (fr) Composés d'aminopyrimidinyle en tant qu'inhibiteurs de jak
LTPA2020510I1 (lt) Pakeistieji 4-fenil-piridinai, skirti su NK-receptoriumi susijusių ligų gydymui
DK3636764T3 (da) 3'-utr-sekvenser til rna-stabilisering
MA44050A (fr) Nouveaux composés
TR201906366T4 (tr) Polisiklik-karbamoilpiridon bileşiklerinin sentezi.
DK3350163T3 (da) Mono- eller disubstituerede indolderivater som hæmmere af denguevirusreplikation
DK3259349T3 (da) Dehydrogenase-katalyseret fremstilling af FDCA
DK3261726T3 (da) Sammensætning til behandling af acne
CU20180001A7 (es) Derivados de oxopiridina sustituidos y procesos para su preparación
DK3359522T3 (da) Proces til fremstilling af N-Boc-bifenylalaninol
DK3224268T3 (da) Nye 2'- og/eller 5'-aminosyreesterphosphoramidat 3'-deoxyadenosinderivater som anti-cancerforbindelser
DK3445850T3 (da) Fremgangsmåder til tilvejebringelse af enkeltstrenget rna
DK3283511T3 (da) Modified collagen, methods of manufacture thereof
DK3130592T3 (da) ANALOGER AF 4H-PYRAZOLO[1,5-á]BENZIMIDAZOL-FORBINDELSER SOM PARPINHIBITORER
DK3356329T3 (da) Fremgangsmåde til fremstilling af 1-(4-methansulfonyl-2-trifluormethylbenzyl)-2-methyl-1H-pyrrolo[2,3-B]pyridin-3-yleddikesyre
FR3019967B1 (fr) Procede d'ostreiculture ameliore
ES2883282T3 (es) Tratamiento de oligoovulación relacionada con esteatosis hepática
DK3302573T3 (da) Sammensætning til behandlingen af hjernelæsioner
MX2017010901A (es) Colorantes de acido trisazo.
FR3038312B1 (fr) Procede de preparation de polyols
DK3351633T3 (da) Antisense-nukleinsyre
DK3145553T3 (da) Lille interfererende rna (sirna) til behandling af type 2 (ado2) autosomal dominant osteopetrose forårsaget af clcn7 (ado2 clcn7-afhængig)-genmutation
DK3317400T3 (da) Fremgangsmåde til kryokonservering af tumorinfiltrerende lymfocytter
ITUA20161990A1 (it) Mangiatoia per avicoltura
ITMI20140333U1 (it) Struttura di porta-disco, dotata di raschia-dischi, particolarmente per erpici o frangizolle