CA3221707A1 - Regulatory nucleic acid sequences - Google Patents
Regulatory nucleic acid sequences Download PDFInfo
- Publication number
- CA3221707A1 CA3221707A1 CA3221707A CA3221707A CA3221707A1 CA 3221707 A1 CA3221707 A1 CA 3221707A1 CA 3221707 A CA3221707 A CA 3221707A CA 3221707 A CA3221707 A CA 3221707A CA 3221707 A1 CA3221707 A1 CA 3221707A1
- Authority
- CA
- Canada
- Prior art keywords
- muscle
- promoter
- specific
- synthetic
- gene
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
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
- C12N15/63—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
-
- 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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2108997.4A GB202108997D0 (en) | 2021-06-23 | 2021-06-23 | Regulatory nucleic acid sequences |
| GB2108997.4 | 2021-06-23 | ||
| GB2111317.0 | 2021-08-05 | ||
| GB202111317 | 2021-08-05 | ||
| PCT/GB2022/051611 WO2022269269A1 (en) | 2021-06-23 | 2022-06-23 | Regulatory nucleic acid sequences |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3221707A1 true CA3221707A1 (en) | 2022-12-29 |
Family
ID=82655313
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3221707A Pending CA3221707A1 (en) | 2021-06-23 | 2022-06-23 | Regulatory nucleic acid sequences |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20250129381A1 (https=) |
| EP (1) | EP4359548A1 (https=) |
| JP (1) | JP2024524270A (https=) |
| KR (1) | KR20240023643A (https=) |
| AU (1) | AU2022297795A1 (https=) |
| CA (1) | CA3221707A1 (https=) |
| IL (1) | IL309630A (https=) |
| WO (1) | WO2022269269A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB202319008D0 (en) * | 2023-12-13 | 2024-01-24 | Ucl Business Ltd | AAV-mediated gene therapy of diseases caused by impaired function of GLDC |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4683195A (en) | 1986-01-30 | 1987-07-28 | Cetus Corporation | Process for amplifying, detecting, and/or-cloning nucleic acid sequences |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US8106180B2 (en) | 2003-08-07 | 2012-01-31 | Whitehead Institute For Biomedical Research | Methods and products for expression of micro RNAs |
| US20050142581A1 (en) | 2003-09-04 | 2005-06-30 | Griffey Richard H. | Microrna as ligands and target molecules |
| WO2005056761A2 (en) | 2003-12-05 | 2005-06-23 | Massachusetts Institute Of Technology | REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi |
| KR100614827B1 (ko) | 2004-05-06 | 2006-08-25 | 재단법인서울대학교산학협력재단 | 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체 |
| PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| TWI908746B (zh) * | 2019-11-28 | 2025-12-21 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
| EP4081643A1 (en) * | 2019-12-24 | 2022-11-02 | Asklepios Biopharmaceutical, Inc. | Regulatory nucleic acid sequences |
| EP4192580A4 (en) * | 2020-08-05 | 2025-06-18 | AskBio Inc. | Methods for treating heart disease and congestive heart failure and administration of AAV vectors |
| CA3195177A1 (en) * | 2020-10-07 | 2022-04-14 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i) |
-
2022
- 2022-06-23 AU AU2022297795A patent/AU2022297795A1/en active Pending
- 2022-06-23 WO PCT/GB2022/051611 patent/WO2022269269A1/en not_active Ceased
- 2022-06-23 KR KR1020247002456A patent/KR20240023643A/ko active Pending
- 2022-06-23 US US18/572,668 patent/US20250129381A1/en active Pending
- 2022-06-23 JP JP2023579317A patent/JP2024524270A/ja active Pending
- 2022-06-23 EP EP22744500.4A patent/EP4359548A1/en active Pending
- 2022-06-23 IL IL309630A patent/IL309630A/en unknown
- 2022-06-23 CA CA3221707A patent/CA3221707A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024524270A (ja) | 2024-07-05 |
| WO2022269269A1 (en) | 2022-12-29 |
| EP4359548A1 (en) | 2024-05-01 |
| KR20240023643A (ko) | 2024-02-22 |
| IL309630A (en) | 2024-02-01 |
| AU2022297795A1 (en) | 2024-01-04 |
| US20250129381A1 (en) | 2025-04-24 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| P22 | Classification modified |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P22-P110 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CLASSIFICATION MODIFIED Effective date: 20241022 |
|
| R11 | Change to the name of applicant or owner or transfer of ownership requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R11-R103 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUEST RECEIVED Effective date: 20250109 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250110 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD Year of fee payment: 3 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250613 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250613 |
|
| R13 | Change to the name of applicant or owner recorded |
Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R13-R104 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250619 |