PL3145553T3 - Mały interferujący RNA (siRNA) do leczenia autosomalnej, dominującej osteopetrozy spowodowanej mutacją genu CLCN7 (ADO2 zależnej od CLCN7) - Google Patents
Mały interferujący RNA (siRNA) do leczenia autosomalnej, dominującej osteopetrozy spowodowanej mutacją genu CLCN7 (ADO2 zależnej od CLCN7)Info
- Publication number
- PL3145553T3 PL3145553T3 PL15732387T PL15732387T PL3145553T3 PL 3145553 T3 PL3145553 T3 PL 3145553T3 PL 15732387 T PL15732387 T PL 15732387T PL 15732387 T PL15732387 T PL 15732387T PL 3145553 T3 PL3145553 T3 PL 3145553T3
- Authority
- PL
- Poland
- Prior art keywords
- ado2
- clcn7
- sirna
- therapy
- dependent
- Prior art date
Links
- 101100001085 Dictyostelium discoideum ado-2 gene Proteins 0.000 title 2
- 108020004459 Small interfering RNA Proteins 0.000 title 2
- 206010064571 Gene mutation Diseases 0.000 title 1
- 208000030757 autosomal dominant osteopetrosis Diseases 0.000 title 1
- 230000001419 dependent effect Effects 0.000 title 1
- 239000004055 small Interfering RNA Substances 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
Classifications
-
- 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 nucleic acids [NA]
-
- 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
Landscapes
- 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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (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)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| ITRM20140272 | 2014-05-23 | ||
| EP15732387.4A EP3145553B1 (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 |
| 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 |
|---|---|
| PL3145553T3 true PL3145553T3 (pl) | 2020-03-31 |
Family
ID=51220775
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL15732387T PL3145553T3 (pl) | 2014-05-23 | 2015-05-21 | Mały interferujący RNA (siRNA) do leczenia autosomalnej, dominującej osteopetrozy spowodowanej mutacją genu CLCN7 (ADO2 zależnej od CLCN7) |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US10450571B2 (pl) |
| EP (1) | EP3145553B1 (pl) |
| JP (1) | JP6795492B2 (pl) |
| AU (1) | AU2015262889B2 (pl) |
| CA (1) | CA2949345C (pl) |
| DK (1) | DK3145553T3 (pl) |
| ES (1) | ES2753355T3 (pl) |
| HU (1) | HUE046546T2 (pl) |
| PL (1) | PL3145553T3 (pl) |
| PT (1) | PT3145553T (pl) |
| WO (1) | WO2015177743A1 (pl) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3029602A1 (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)
| 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 |
-
2015
- 2015-05-21 EP EP15732387.4A patent/EP3145553B1/en active Active
- 2015-05-21 PT PT157323874T patent/PT3145553T/pt unknown
- 2015-05-21 WO PCT/IB2015/053730 patent/WO2015177743A1/en not_active Ceased
- 2015-05-21 PL PL15732387T patent/PL3145553T3/pl unknown
- 2015-05-21 DK DK15732387T patent/DK3145553T3/da active
- 2015-05-21 JP JP2017513379A patent/JP6795492B2/ja active Active
- 2015-05-21 CA CA2949345A patent/CA2949345C/en active Active
- 2015-05-21 US US15/313,221 patent/US10450571B2/en active Active
- 2015-05-21 AU AU2015262889A patent/AU2015262889B2/en active Active
- 2015-05-21 ES ES15732387T patent/ES2753355T3/es active Active
- 2015-05-21 HU HUE15732387A patent/HUE046546T2/hu unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3145553B1 (en) | 2019-07-31 |
| AU2015262889A8 (en) | 2017-02-02 |
| HUE046546T2 (hu) | 2020-03-30 |
| ES2753355T3 (es) | 2020-04-08 |
| CA2949345C (en) | 2022-06-14 |
| JP2017521094A (ja) | 2017-08-03 |
| AU2015262889A1 (en) | 2016-12-01 |
| AU2015262889B2 (en) | 2020-04-23 |
| US20170101644A1 (en) | 2017-04-13 |
| CA2949345A1 (en) | 2015-11-26 |
| DK3145553T3 (da) | 2019-10-28 |
| PT3145553T (pt) | 2019-10-30 |
| US10450571B2 (en) | 2019-10-22 |
| EP3145553A1 (en) | 2017-03-29 |
| WO2015177743A1 (en) | 2015-11-26 |
| JP6795492B2 (ja) | 2020-12-02 |
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