MX2020008533A - Terapia con oligonucleotidos para la enfermedad de wilson. - Google Patents
Terapia con oligonucleotidos para la enfermedad de wilson.Info
- Publication number
- MX2020008533A MX2020008533A MX2020008533A MX2020008533A MX2020008533A MX 2020008533 A MX2020008533 A MX 2020008533A MX 2020008533 A MX2020008533 A MX 2020008533A MX 2020008533 A MX2020008533 A MX 2020008533A MX 2020008533 A MX2020008533 A MX 2020008533A
- Authority
- MX
- Mexico
- Prior art keywords
- exon
- atp7b
- flanking
- wilson disease
- flanking intron
- Prior art date
Links
Classifications
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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
- C12N15/1137—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/33—Alteration of splicing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
- C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (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)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
La presente invención se refiere a oligonucleótidos antisentido, composiciones y métodos que se dirigen al exón 6 de ATP7B o un intrón flanqueante, modulando así el empalme de pre-ARNm de ATP7B para aumentar el nivel de moléculas de ARNm de ATP7B que tienen exón 6, por ejemplo, para proporcionar una terapia para la enfermedad de Wilson. La presente descripción proporciona un oligonucleótido antisentido que incluye una secuencia de nucleobase al menos 70% complementaria a una secuencia objetivo de ATP7B en el exón 6, un intrón flanqueante en 5', un intrón flanqueante en 3' o una combinación del exón 6 y el intrón flanqueante en 5' o flanqueante en 3'.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862630565P | 2018-02-14 | 2018-02-14 | |
PCT/US2019/018076 WO2019161105A1 (en) | 2018-02-14 | 2019-02-14 | Oligonucleotide therapy for wilson disease |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020008533A true MX2020008533A (es) | 2020-11-06 |
Family
ID=67619598
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020008533A MX2020008533A (es) | 2018-02-14 | 2019-02-14 | Terapia con oligonucleotidos para la enfermedad de wilson. |
Country Status (14)
Country | Link |
---|---|
US (1) | US11578327B2 (es) |
EP (1) | EP3752614A4 (es) |
JP (1) | JP2021513851A (es) |
KR (1) | KR20200120675A (es) |
CN (1) | CN112041439A (es) |
AU (1) | AU2019222767A1 (es) |
BR (1) | BR112020016524A2 (es) |
CA (1) | CA3090517A1 (es) |
CL (1) | CL2020002100A1 (es) |
CO (1) | CO2020010226A2 (es) |
IL (1) | IL276701A (es) |
MX (1) | MX2020008533A (es) |
SG (1) | SG11202007644PA (es) |
WO (1) | WO2019161105A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
Family Cites Families (36)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
US5578493A (en) * | 1993-09-01 | 1996-11-26 | The Trustees Of Columbia University In The City Of New York | Wilson's disease gene |
CA2108927C (en) | 1993-09-21 | 2008-09-02 | Peter Bull | Wilson disease gene |
CN1120707C (zh) | 1995-11-22 | 2003-09-10 | 约翰斯·霍普金斯大学 | 增强生物分子的细胞摄取的配体 |
US20040101854A1 (en) * | 2002-11-21 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of BCL2-associated athanogene expression |
US7491805B2 (en) | 2001-05-18 | 2009-02-17 | Sirna Therapeutics, Inc. | Conjugates and compositions for cellular delivery |
US20030180953A1 (en) | 2000-12-29 | 2003-09-25 | Elitra Pharmaceuticals, Inc. | Gene disruption methodologies for drug target discovery |
US20040126761A1 (en) * | 2002-12-10 | 2004-07-01 | Isis Pharmaceuticals Inc. | Modulation of alpha-methylacyl-CoA racemase expression |
AU2003273778A1 (en) | 2002-10-21 | 2004-05-04 | Exiqon A/S | Oligonucleotide analogues for detecting and analyzing nucleic acids |
US20050244851A1 (en) | 2004-01-13 | 2005-11-03 | Affymetrix, Inc. | Methods of analysis of alternative splicing in human |
US20070280927A1 (en) * | 2004-01-29 | 2007-12-06 | Cellzome Ag | Treatment Of Neurodegenerative Diseases By The Use Of Atp7a |
AU2005248147A1 (en) | 2004-05-11 | 2005-12-08 | Alphagen Co., Ltd. | Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same |
EP1784501B1 (en) | 2004-05-14 | 2015-11-18 | Rosetta Genomics Ltd | VIRAL AND VIRUS ASSOCIATED MicroRNAS AND USES THEREOF |
US20070048756A1 (en) | 2005-04-18 | 2007-03-01 | Affymetrix, Inc. | Methods for whole genome association studies |
EP1783645A1 (en) * | 2005-11-08 | 2007-05-09 | Actigenics | Methods for the identification of microRNA and their applications in research and human health |
US8178503B2 (en) | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
JP4841279B2 (ja) | 2006-03-23 | 2011-12-21 | 一般財団法人 化学物質評価研究機構 | 被検物質の発がん性予測方法 |
CA2708153C (en) | 2007-12-04 | 2017-09-26 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
EP2802658A2 (en) * | 2012-01-09 | 2014-11-19 | Novartis AG | Rnai agents to treat beta-catenin related diseases |
WO2014036301A1 (en) * | 2012-08-30 | 2014-03-06 | Isis Pharmaceuticals, Inc. | Modulation of copper related diseases by ctr1 |
EP2991661B1 (en) | 2013-05-01 | 2019-03-13 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
WO2015002978A2 (en) * | 2013-07-02 | 2015-01-08 | The Trustees Of The University Of Pennsylvania | Methods for rapid ribonucleic acid fluorescence in situ hybridization |
US20160257961A1 (en) | 2013-11-06 | 2016-09-08 | Solstice Biologics, Ltd. | Polynucleotide constructs having disulfide groups |
CN107109405A (zh) | 2014-06-06 | 2017-08-29 | 索尔斯蒂斯生物有限公司 | 具有生物可逆性和非生物可逆性基团的多核苷酸构建体 |
WO2016004043A1 (en) | 2014-06-30 | 2016-01-07 | Blend Therapeutics, Inc. | Targeted conjugates and particles and formulations thereof |
US10822369B2 (en) | 2014-11-14 | 2020-11-03 | Ionis Pharmaceuticals, Inc. | Compounds and methods for the modulation of proteins |
EP3247366A4 (en) | 2015-01-21 | 2018-10-31 | Sangamo Therapeutics, Inc. | Methods and compositions for identification of highly specific nucleases |
CN105986015B (zh) * | 2015-02-05 | 2020-10-23 | 大连晶泰生物技术有限公司 | 一种基于高通量测序的多样本的一个或多个靶序列的检测方法和试剂盒 |
WO2016170348A2 (en) | 2015-04-22 | 2016-10-27 | Mina Therapeutics Limited | Sarna compositions and methods of use |
WO2017100461A1 (en) | 2015-12-08 | 2017-06-15 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
CA3005090A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
KR101743211B1 (ko) | 2016-06-24 | 2017-06-15 | 주식회사 녹십자지놈 | 선천성 기능장애 진단용 조성물 및 이의 용도 |
EP3840758A1 (en) * | 2018-08-21 | 2021-06-30 | Deep Genomics Incorporated | Therapeutic splice-switching oligonucleotides |
CA3218805A1 (en) * | 2021-05-10 | 2022-11-17 | Ziqing QIAN | Compositions and methods for intracellular therapeutics |
WO2022256283A2 (en) * | 2021-06-01 | 2022-12-08 | Korro Bio, Inc. | Methods for restoring protein function using adar |
-
2019
- 2019-02-14 MX MX2020008533A patent/MX2020008533A/es unknown
- 2019-02-14 EP EP19754072.7A patent/EP3752614A4/en active Pending
- 2019-02-14 BR BR112020016524-7A patent/BR112020016524A2/pt not_active IP Right Cessation
- 2019-02-14 AU AU2019222767A patent/AU2019222767A1/en not_active Abandoned
- 2019-02-14 JP JP2020543576A patent/JP2021513851A/ja not_active Withdrawn
- 2019-02-14 KR KR1020207025862A patent/KR20200120675A/ko unknown
- 2019-02-14 CN CN201980018495.1A patent/CN112041439A/zh active Pending
- 2019-02-14 WO PCT/US2019/018076 patent/WO2019161105A1/en unknown
- 2019-02-14 US US16/970,088 patent/US11578327B2/en active Active
- 2019-02-14 CA CA3090517A patent/CA3090517A1/en active Pending
- 2019-02-14 SG SG11202007644PA patent/SG11202007644PA/en unknown
-
2020
- 2020-08-13 IL IL276701A patent/IL276701A/en unknown
- 2020-08-13 CL CL2020002100A patent/CL2020002100A1/es unknown
- 2020-08-19 CO CONC2020/0010226A patent/CO2020010226A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
AU2019222767A1 (en) | 2020-08-27 |
US11578327B2 (en) | 2023-02-14 |
JP2021513851A (ja) | 2021-06-03 |
CO2020010226A2 (es) | 2020-12-10 |
CN112041439A (zh) | 2020-12-04 |
WO2019161105A1 (en) | 2019-08-22 |
IL276701A (en) | 2020-09-30 |
EP3752614A4 (en) | 2021-11-10 |
BR112020016524A2 (pt) | 2021-09-28 |
KR20200120675A (ko) | 2020-10-21 |
CL2020002100A1 (es) | 2021-01-29 |
CA3090517A1 (en) | 2019-08-22 |
EP3752614A1 (en) | 2020-12-23 |
US20210032629A1 (en) | 2021-02-04 |
SG11202007644PA (en) | 2020-09-29 |
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