PL4154914T3 - Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana - Google Patents
Zmodyfikowany gen ube3a w terapii genowej zespołu angelmanaInfo
- Publication number
- PL4154914T3 PL4154914T3 PL22186588.4T PL22186588T PL4154914T3 PL 4154914 T3 PL4154914 T3 PL 4154914T3 PL 22186588 T PL22186588 T PL 22186588T PL 4154914 T3 PL4154914 T3 PL 4154914T3
- Authority
- PL
- Poland
- Prior art keywords
- gene
- angelman syndrome
- therapy approach
- gene therapy
- modified
- Prior art date
Links
- 208000009575 Angelman syndrome Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
Classifications
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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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P15/00—Drugs for genital or sexual disorders; Contraceptives
- A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/04—Anorexiants; Antiobesity agents
-
- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
-
- 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
- C12N9/10—Transferases (2.)
- C12N9/1025—Acyltransferases (2.3)
- C12N9/104—Aminoacyltransferases (2.3.2)
-
- 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
- C12N9/93—Ligases (6)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y203/00—Acyltransferases (2.3)
- C12Y203/02—Aminoacyltransferases (2.3.2)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
- C12Y603/02—Acid—amino-acid ligases (peptide synthases)(6.3.2)
- C12Y603/02019—Ubiquitin-protein ligase (6.3.2.19), i.e. ubiquitin-conjugating enzyme
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/036—Fusion polypeptide containing a localisation/targetting motif targeting to the medium outside of the cell, e.g. type III secretion
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Reproductive Health (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Gynecology & Obstetrics (AREA)
- Pregnancy & Childbirth (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Child & Adolescent Psychology (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562158269P | 2015-05-07 | 2015-05-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL4154914T3 true PL4154914T3 (pl) | 2025-01-07 |
Family
ID=57218344
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL22186588.4T PL4154914T3 (pl) | 2015-05-07 | 2016-05-09 | Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana |
| PL16790226.1T PL3291843T3 (pl) | 2015-05-07 | 2016-05-09 | Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL16790226.1T PL3291843T3 (pl) | 2015-05-07 | 2016-05-09 | Zmodyfikowany gen ube3a w terapii genowej zespołu angelmana |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11534500B2 (pl) |
| EP (2) | EP3291843B1 (pl) |
| JP (1) | JP6841507B2 (pl) |
| CN (1) | CN107530451A (pl) |
| CA (1) | CA2984629C (pl) |
| ES (2) | ES2947311T3 (pl) |
| HU (2) | HUE068547T2 (pl) |
| PL (2) | PL4154914T3 (pl) |
| WO (1) | WO2016179584A1 (pl) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20200138921A1 (en) * | 2017-06-23 | 2020-05-07 | The Trustees Of Columbia University In The City Of New York | Methods of preventing and treating diseases characterized by synaptic dysfunction and neurodegeneration including alzheimer's disease |
| BR112019027692A2 (pt) * | 2017-06-28 | 2020-11-24 | University Of South Florida | gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman |
| CN110237257B (zh) * | 2018-03-09 | 2023-01-03 | 中国科学院脑科学与智能技术卓越创新中心 | Ube3a泛素化PP2A激活因子PTPA在治疗天使综合症和孤独症中的应用 |
| KR20210040358A (ko) * | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
| EP3911957A1 (en) * | 2019-01-17 | 2021-11-24 | F. Hoffmann-La Roche AG | E3 ubiquitin ligase (ube3a) protein targets |
| WO2020159965A1 (en) * | 2019-01-30 | 2020-08-06 | University Of South Florida | Method for detection and analysis of cerebrospinal fluid associated ube3a |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| US12465655B2 (en) * | 2019-05-22 | 2025-11-11 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| IT201900008877A1 (it) * | 2019-06-13 | 2020-12-13 | Univ Bologna Alma Mater Studiorum | Nuovi costrutti per terapia genica |
| EP4017523A4 (en) * | 2019-08-22 | 2024-01-17 | The Regents Of The University Of California | UBE3A FOR THE TREATMENT OF ANGELMAN SYNDROME |
| JP2023524247A (ja) * | 2020-04-28 | 2023-06-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アンジェルマン症候群の治療のための組成物及びその使用 |
| WO2022272171A2 (en) * | 2021-06-25 | 2022-12-29 | University Of South Florida | Secreted ube3a for treatment of neurological disorders |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5658785A (en) * | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| AUPP655698A0 (en) * | 1998-10-16 | 1998-11-05 | Commonwealth Scientific And Industrial Research Organisation | Delivery system for porcine somatotropin |
| US6706505B1 (en) * | 2000-03-08 | 2004-03-16 | Amgen Inc | Human E3α ubiquitin ligase family |
| AU2001257078A1 (en) * | 2000-04-18 | 2001-10-30 | Iconix Pharmaceuticals, Inc. | Vector and method for targeted replacement and disruption of an integrated dna sequence |
| WO2001092582A1 (en) * | 2000-06-01 | 2001-12-06 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the ube3a gene |
| JP2006506044A (ja) * | 2002-04-19 | 2006-02-23 | ジョージア テック リサーチ コーポレイション | タンパク質分泌ダイナミクスの加速のための組成物及び方法 |
| NZ544263A (en) * | 2003-06-10 | 2009-04-30 | Nsgene As | Improved secretion of neublastin |
| MXPA06007574A (es) * | 2003-12-31 | 2007-04-17 | Sanofi Pasteur Inc | Inmunogenos dirigidos. |
| US20050271625A1 (en) * | 2004-03-02 | 2005-12-08 | Nash Kevin R | RAAV-neprilysin compositions and methods of use |
| WO2011109398A2 (en) | 2010-03-02 | 2011-09-09 | President And Fellows Of Harvard College | Methods and compositions for treatment of angelman syndrome and autism spectrum disorders |
| US9714427B2 (en) | 2010-11-11 | 2017-07-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| PT3358011T (pt) * | 2011-04-26 | 2020-04-23 | Univ Osaka | Péptido para induzir a regeneração de um tecido e a sua utilização |
| WO2013016279A1 (en) * | 2011-07-25 | 2013-01-31 | Beth Israel Deaconess Medical Center, Inc. | Animal model of autism |
| WO2014004572A2 (en) | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| EP2724721A1 (en) | 2012-10-26 | 2014-04-30 | Matentzoglu, Konstantin | Composition for use in the treatment of Angelman syndrome and/or autism spectrum disorder, the use of such composition and a method for manufacturing a medicament for the treatment of Angelman syndrome and/or autism spectrum disorder |
| US11053291B2 (en) | 2014-02-19 | 2021-07-06 | University Of Florida Research Foundation, Incorporated | Delivery of Nrf2 as therapy for protection against reactive oxygen species |
| EP3155003B9 (en) * | 2014-06-13 | 2020-12-30 | Whitehead Institute for Biomedical Research | Amyloid beta expression constructs and uses therefor |
-
2016
- 2016-05-09 EP EP16790226.1A patent/EP3291843B1/en active Active
- 2016-05-09 CN CN201680026461.3A patent/CN107530451A/zh active Pending
- 2016-05-09 PL PL22186588.4T patent/PL4154914T3/pl unknown
- 2016-05-09 WO PCT/US2016/031468 patent/WO2016179584A1/en not_active Ceased
- 2016-05-09 EP EP22186588.4A patent/EP4154914B1/en active Active
- 2016-05-09 PL PL16790226.1T patent/PL3291843T3/pl unknown
- 2016-05-09 HU HUE22186588A patent/HUE068547T2/hu unknown
- 2016-05-09 HU HUE16790226A patent/HUE062186T2/hu unknown
- 2016-05-09 CA CA2984629A patent/CA2984629C/en active Active
- 2016-05-09 ES ES16790226T patent/ES2947311T3/es active Active
- 2016-05-09 JP JP2017556744A patent/JP6841507B2/ja active Active
- 2016-05-09 ES ES22186588T patent/ES2992293T3/es active Active
-
2017
- 2017-11-07 US US15/805,442 patent/US11534500B2/en active Active
-
2022
- 2022-12-23 US US18/087,885 patent/US20230277684A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US11534500B2 (en) | 2022-12-27 |
| CA2984629C (en) | 2024-06-18 |
| HUE062186T2 (hu) | 2023-09-28 |
| EP3291843A1 (en) | 2018-03-14 |
| JP6841507B2 (ja) | 2021-03-10 |
| PL3291843T3 (pl) | 2023-07-17 |
| US20180104358A1 (en) | 2018-04-19 |
| HUE068547T2 (hu) | 2025-01-28 |
| EP3291843B1 (en) | 2023-03-22 |
| CA2984629A1 (en) | 2016-11-10 |
| JP2018518946A (ja) | 2018-07-19 |
| CN107530451A (zh) | 2018-01-02 |
| EP3291843A4 (en) | 2018-12-05 |
| EP4154914A1 (en) | 2023-03-29 |
| ES2947311T3 (es) | 2023-08-04 |
| ES2992293T3 (es) | 2024-12-11 |
| WO2016179584A1 (en) | 2016-11-10 |
| EP4154914B1 (en) | 2024-08-28 |
| US20230277684A1 (en) | 2023-09-07 |
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