SG11202105030VA - Method for treating muscular dystrophy by targeting utrophin gene - Google Patents
Method for treating muscular dystrophy by targeting utrophin geneInfo
- Publication number
- SG11202105030VA SG11202105030VA SG11202105030VA SG11202105030VA SG11202105030VA SG 11202105030V A SG11202105030V A SG 11202105030VA SG 11202105030V A SG11202105030V A SG 11202105030VA SG 11202105030V A SG11202105030V A SG 11202105030VA SG 11202105030V A SG11202105030V A SG 11202105030VA
- Authority
- SG
- Singapore
- Prior art keywords
- targeting
- muscular dystrophy
- treating muscular
- utrophin gene
- utrophin
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
-
- 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
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- A—HUMAN NECESSITIES
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
-
- 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
- C12N15/63—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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- 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
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862768474P | 2018-11-16 | 2018-11-16 | |
| US201962861039P | 2019-06-13 | 2019-06-13 | |
| US201962931925P | 2019-11-07 | 2019-11-07 | |
| PCT/JP2019/045716 WO2020101042A1 (en) | 2018-11-16 | 2019-11-15 | Method for treating muscular dystrophy by targeting utrophin gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11202105030VA true SG11202105030VA (en) | 2021-06-29 |
Family
ID=68988228
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202105030VA SG11202105030VA (en) | 2018-11-16 | 2019-11-15 | Method for treating muscular dystrophy by targeting utrophin gene |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US11473071B2 (en) |
| EP (1) | EP3880255A1 (en) |
| JP (2) | JP7069426B2 (en) |
| KR (1) | KR20210093954A (en) |
| CN (1) | CN113271982A (en) |
| AU (1) | AU2019381460A1 (en) |
| BR (1) | BR112021009481A2 (en) |
| CA (1) | CA3119618A1 (en) |
| CO (1) | CO2021006957A2 (en) |
| IL (1) | IL283123A (en) |
| MX (1) | MX2021005720A (en) |
| PH (1) | PH12021551122A1 (en) |
| SG (1) | SG11202105030VA (en) |
| TW (1) | TW202033224A (en) |
| WO (1) | WO2020101042A1 (en) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102008496B1 (en) * | 2013-03-25 | 2019-08-07 | 삼성전자주식회사 | Method and apparatus for detecting direction and velocity of equipment using a single sensor |
| CA3096713A1 (en) | 2018-04-19 | 2019-10-24 | The Regents Of The University Of California | Compositions and methods for gene editing |
| JP7412001B2 (en) * | 2018-06-08 | 2024-01-12 | 株式会社モダリス | Modified Cas9 protein and its uses |
| BR112021002231A2 (en) * | 2018-08-07 | 2021-05-04 | Modalis Therapeutics Corporation | new transcription activator |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| CA3200563A1 (en) | 2020-09-29 | 2022-04-07 | Genethon | Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof |
| EP4331579A1 (en) | 2021-04-26 | 2024-03-06 | KSB Tugen Inc. | Composition comprising oxiracetam for preventing or treating muscular disease |
| KR102421411B1 (en) | 2021-10-29 | 2022-07-18 | 주식회사 케이에스비튜젠 | Composition for preventing or treating Muscular Dystrophy comprising Oxiracetam |
| CN113563428B (en) * | 2021-06-22 | 2022-08-02 | 呈诺再生医学科技(珠海横琴新区)有限公司 | Polypeptide with specific spatial structure and application thereof in preparation of iPSC |
| WO2023190935A1 (en) * | 2022-03-31 | 2023-10-05 | Modalis Therapeutics Corporation | Method for treating myopathies by targeting titin gene |
| CN116218849B (en) * | 2023-01-18 | 2024-01-23 | 昆明理工大学 | sgRNA for treating Dunaliella muscular dystrophy by activating utrophin and application |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1287125B1 (en) * | 2000-04-28 | 2009-07-29 | Asklepios Biopharmaceutical, Inc. | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| CA2448120A1 (en) | 2001-05-24 | 2002-11-28 | Genzyme Corporation | Muscle-specific expression vectors |
| CN105671005B (en) | 2001-11-13 | 2020-09-15 | 宾夕法尼亚大学托管会 | Methods for detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified |
| DK2292779T3 (en) | 2003-09-30 | 2017-02-27 | Univ Pennsylvania | ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF |
| CA2553676A1 (en) | 2004-01-28 | 2005-08-11 | Research Development Foundation | Human soluble neuropilin-1 primary polyadenylation signal and uses thereof |
| ITRM20070523A1 (en) | 2007-10-05 | 2009-04-06 | Consiglio Nazionale Ricerche | CODIFYING NUCLEIC ACID FOR A REGULATING PROTEIN SPECIFIC TO THE TRANSCRIPTION OF THE UROPHINE PROTEIN BY IT CODIFIED AND ITS APPLICATIONS |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2013173645A1 (en) * | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating utrn expression |
| DK3241902T3 (en) | 2012-05-25 | 2018-05-07 | Univ California | METHODS AND COMPOSITIONS FOR RNA DIRECTIVE TARGET DNA MODIFICATION AND FOR RNA DIRECTIVE MODULATION OF TRANSCRIPTION |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| ITTO20130669A1 (en) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | ADENO-ASSOCIATED MOMCULAR-SPECIFIC VECTOR AND ITS EMPLOYMENT IN THE TREATMENT OF MUSCLE PATHOLOGIES |
| AU2016237148A1 (en) | 2015-03-24 | 2017-11-23 | Kyowa Hakko Kirin Co., Ltd. | Nucleic acid-containing lipid nanoparticles |
| CA2996982A1 (en) * | 2015-09-23 | 2017-03-30 | Universite Laval | Modification of the dystrophin gene and uses thereof |
| EP3413908A4 (en) * | 2016-02-11 | 2019-09-25 | The Regents of The University of California | Methods and compositions for modifying a mutant dystrophin gene in a cell's genome |
| US11674128B2 (en) * | 2016-12-12 | 2023-06-13 | Tsinghua University | Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA |
| CN110612113B (en) * | 2017-02-07 | 2024-03-26 | 加利福尼亚大学董事会 | Gene therapy for haploid insufficiency |
| US11674138B2 (en) * | 2017-03-13 | 2023-06-13 | President And Fellows Of Harvard College | Methods of modulating expression of target nucleic acid sequences in a cell |
| US20210254056A1 (en) * | 2017-05-05 | 2021-08-19 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| CA3097375A1 (en) | 2018-04-27 | 2019-10-31 | Universitat Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| WO2019236081A1 (en) * | 2018-06-06 | 2019-12-12 | Salk Institute For Biological Studies | Targeted gene activation using modified guide rna |
-
2019
- 2019-11-15 SG SG11202105030VA patent/SG11202105030VA/en unknown
- 2019-11-15 AU AU2019381460A patent/AU2019381460A1/en not_active Abandoned
- 2019-11-15 JP JP2021551108A patent/JP7069426B2/en active Active
- 2019-11-15 CA CA3119618A patent/CA3119618A1/en active Pending
- 2019-11-15 KR KR1020217018333A patent/KR20210093954A/en unknown
- 2019-11-15 CN CN201980075173.0A patent/CN113271982A/en active Pending
- 2019-11-15 EP EP19824401.4A patent/EP3880255A1/en active Pending
- 2019-11-15 MX MX2021005720A patent/MX2021005720A/en unknown
- 2019-11-15 WO PCT/JP2019/045716 patent/WO2020101042A1/en unknown
- 2019-11-15 TW TW108141671A patent/TW202033224A/en unknown
- 2019-11-15 BR BR112021009481-4A patent/BR112021009481A2/en unknown
-
2021
- 2021-05-11 PH PH12021551122A patent/PH12021551122A1/en unknown
- 2021-05-12 IL IL283123A patent/IL283123A/en unknown
- 2021-05-14 US US17/320,643 patent/US11473071B2/en active Active
- 2021-05-26 CO CONC2021/0006957A patent/CO2021006957A2/en unknown
-
2022
- 2022-05-02 JP JP2022076261A patent/JP2022115976A/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3119618A1 (en) | 2020-05-22 |
| KR20210093954A (en) | 2021-07-28 |
| CN113271982A (en) | 2021-08-17 |
| PH12021551122A1 (en) | 2021-11-22 |
| IL283123A (en) | 2021-06-30 |
| US20210355464A1 (en) | 2021-11-18 |
| JP7069426B2 (en) | 2022-05-17 |
| AU2019381460A1 (en) | 2021-06-17 |
| MX2021005720A (en) | 2021-07-21 |
| CO2021006957A2 (en) | 2021-06-10 |
| BR112021009481A2 (en) | 2021-08-17 |
| WO2020101042A1 (en) | 2020-05-22 |
| US11473071B2 (en) | 2022-10-18 |
| TW202033224A (en) | 2020-09-16 |
| JP2022509560A (en) | 2022-01-20 |
| EP3880255A1 (en) | 2021-09-22 |
| JP2022115976A (en) | 2022-08-09 |
| WO2020101042A8 (en) | 2020-07-30 |
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