CN112469421A - 减少剪接病和治疗rna显性疾病的组合物和方法 - Google Patents
减少剪接病和治疗rna显性疾病的组合物和方法 Download PDFInfo
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- CN112469421A CN112469421A CN201980047374.XA CN201980047374A CN112469421A CN 112469421 A CN112469421 A CN 112469421A CN 201980047374 A CN201980047374 A CN 201980047374A CN 112469421 A CN112469421 A CN 112469421A
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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- 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
- C12N15/1137—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 enzymes
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- C12N9/10—Transferases (2.)
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- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
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- 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]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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]
- C12N2310/141—MicroRNAs, miRNAs
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
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- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
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- Molecular Biology (AREA)
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- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862671769P | 2018-05-15 | 2018-05-15 | |
| US62/671,769 | 2018-05-15 | ||
| PCT/US2019/032423 WO2019222354A1 (en) | 2018-05-15 | 2019-05-15 | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112469421A true CN112469421A (zh) | 2021-03-09 |
Family
ID=68540673
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980047374.XA Pending CN112469421A (zh) | 2018-05-15 | 2019-05-15 | 减少剪接病和治疗rna显性疾病的组合物和方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20210269825A1 (https=) |
| EP (1) | EP3793566A4 (https=) |
| JP (1) | JP2021522836A (https=) |
| KR (1) | KR20210010549A (https=) |
| CN (1) | CN112469421A (https=) |
| AU (1) | AU2019268346A1 (https=) |
| BR (1) | BR112020023298A2 (https=) |
| CA (1) | CA3098249A1 (https=) |
| CL (1) | CL2020002955A1 (https=) |
| CO (1) | CO2020015239A2 (https=) |
| MA (1) | MA51938B1 (https=) |
| MX (1) | MX2020012269A (https=) |
| PH (1) | PH12020551913A1 (https=) |
| SG (1) | SG11202011151VA (https=) |
| WO (1) | WO2019222354A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| TWI900553B (zh) | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| WO2023196862A1 (en) * | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| US20250352672A1 (en) * | 2022-05-13 | 2025-11-20 | University Of Washington | Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting |
| TW202409289A (zh) * | 2022-06-27 | 2024-03-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療肌強直性營養不良之組合物及方法 |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2024229164A2 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025038795A1 (en) * | 2023-08-16 | 2025-02-20 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025122536A1 (en) * | 2023-12-05 | 2025-06-12 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| WO2025259747A2 (en) * | 2024-06-12 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110111491A1 (en) * | 2002-08-05 | 2011-05-12 | University Of Iowa Research Foundation | Rna interference suppresion of neurodegenerative diseases and methods of use thereof |
| CN108064292A (zh) * | 2014-12-24 | 2018-05-22 | 尤尼克尔生物制药股份有限公司 | RNAi诱导的亨廷顿蛋白基因抑制 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5977333A (en) * | 1992-02-06 | 1999-11-02 | Massachusetts Institute Of Technology | DNA sequence encoding the myotonic dystrophy gene and uses thereof |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2595663A4 (en) * | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| WO2017079291A1 (en) * | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| CN115896106A (zh) * | 2016-03-01 | 2023-04-04 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| CN109862785B (zh) * | 2016-09-30 | 2022-09-06 | 瑞泽恩制药公司 | C9orf72基因座中具有六核苷酸重复扩增的非人类动物 |
| CN112292895B (zh) * | 2018-08-10 | 2024-06-14 | 富士通株式会社 | 一种通信方法、装置和通信系统 |
-
2019
- 2019-05-15 BR BR112020023298-0A patent/BR112020023298A2/pt not_active Application Discontinuation
- 2019-05-15 WO PCT/US2019/032423 patent/WO2019222354A1/en not_active Ceased
- 2019-05-15 KR KR1020207036197A patent/KR20210010549A/ko not_active Abandoned
- 2019-05-15 US US17/054,474 patent/US20210269825A1/en not_active Abandoned
- 2019-05-15 MX MX2020012269A patent/MX2020012269A/es unknown
- 2019-05-15 MA MA51938A patent/MA51938B1/fr unknown
- 2019-05-15 JP JP2020563947A patent/JP2021522836A/ja active Pending
- 2019-05-15 AU AU2019268346A patent/AU2019268346A1/en not_active Abandoned
- 2019-05-15 CN CN201980047374.XA patent/CN112469421A/zh active Pending
- 2019-05-15 EP EP19803882.0A patent/EP3793566A4/en not_active Withdrawn
- 2019-05-15 SG SG11202011151VA patent/SG11202011151VA/en unknown
- 2019-05-15 CA CA3098249A patent/CA3098249A1/en active Pending
-
2020
- 2020-11-10 PH PH12020551913A patent/PH12020551913A1/en unknown
- 2020-11-13 CL CL2020002955A patent/CL2020002955A1/es unknown
- 2020-12-03 CO CONC2020/0015239A patent/CO2020015239A2/es unknown
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110111491A1 (en) * | 2002-08-05 | 2011-05-12 | University Of Iowa Research Foundation | Rna interference suppresion of neurodegenerative diseases and methods of use thereof |
| CN108064292A (zh) * | 2014-12-24 | 2018-05-22 | 尤尼克尔生物制药股份有限公司 | RNAi诱导的亨廷顿蛋白基因抑制 |
Non-Patent Citations (3)
| Title |
|---|
| DARRENR.BISSET等: "Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy", 《HUMAN MOLECULAR GENETICS》, vol. 24, no. 17, pages 4971 - 4983, XP055656933, DOI: 10.1093/hmg/ddv219 * |
| JIAXIN HU等: "Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG repeats at the ALS/FTD C9orf72 locus", 《CHEM BIOL. 》, vol. 22, no. 11, pages 1505 - 1511 * |
| MARC-ANDRE LANGLOIS等: "Cytoplasmic and Nuclear Retained DMPK mRNAs Are Targets for RNA Interference in Myotonic Dystrophy Cells", 《THE JOURNAL OF BIOLOGICAL CHEMISTRY》, vol. 280, no. 17, pages 16949 - 16954, XP055147962, DOI: 10.1074/jbc.M501591200 * |
Also Published As
| Publication number | Publication date |
|---|---|
| PH12020551913A1 (en) | 2021-06-14 |
| BR112020023298A2 (pt) | 2021-03-09 |
| EP3793566A4 (en) | 2022-03-16 |
| MA51938A1 (fr) | 2021-11-30 |
| MA51938B1 (fr) | 2022-10-31 |
| WO2019222354A1 (en) | 2019-11-21 |
| MX2020012269A (es) | 2021-04-28 |
| KR20210010549A (ko) | 2021-01-27 |
| US20210269825A1 (en) | 2021-09-02 |
| JP2021522836A (ja) | 2021-09-02 |
| SG11202011151VA (en) | 2020-12-30 |
| EP3793566A1 (en) | 2021-03-24 |
| AU2019268346A1 (en) | 2020-12-24 |
| CO2020015239A2 (es) | 2021-03-08 |
| CA3098249A1 (en) | 2019-11-21 |
| CL2020002955A1 (es) | 2021-04-23 |
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