PL2561067T3 - Kompozycje RAAV-cyklazy guanylanowej i sposoby leczenia wrodzonej amaurozy-1 Lebera (LCA1) - Google Patents
Kompozycje RAAV-cyklazy guanylanowej i sposoby leczenia wrodzonej amaurozy-1 Lebera (LCA1)Info
- Publication number
- PL2561067T3 PL2561067T3 PL11726973T PL11726973T PL2561067T3 PL 2561067 T3 PL2561067 T3 PL 2561067T3 PL 11726973 T PL11726973 T PL 11726973T PL 11726973 T PL11726973 T PL 11726973T PL 2561067 T3 PL2561067 T3 PL 2561067T3
- Authority
- PL
- Poland
- Prior art keywords
- amaurose
- lca1
- leber
- raav
- congenital
- Prior art date
Links
- 206010010356 Congenital anomaly Diseases 0.000 title 1
- 101710095468 Cyclase Proteins 0.000 title 1
- 206010001902 amaurosis Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 238000011282 treatment Methods 0.000 title 1
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
- C12N15/63—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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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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
- C12N15/63—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
- C12N15/861—Adenoviral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/51—Lyases (4)
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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
- C12N15/52—Genes encoding for enzymes or proenzymes
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/88—Lyases (4.)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y406/00—Phosphorus-oxygen lyases (4.6)
- C12Y406/01—Phosphorus-oxygen lyases (4.6.1)
- C12Y406/01002—Guanylate cyclase (4.6.1.2)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- 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
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Ophthalmology & Optometry (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32752110P | 2010-04-23 | 2010-04-23 | |
| EP11726973.8A EP2561067B1 (en) | 2010-04-23 | 2011-04-22 | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
| PCT/US2011/033669 WO2011133933A2 (en) | 2010-04-23 | 2011-04-22 | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2561067T3 true PL2561067T3 (pl) | 2019-05-31 |
Family
ID=44533055
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL11726973T PL2561067T3 (pl) | 2010-04-23 | 2011-04-22 | Kompozycje RAAV-cyklazy guanylanowej i sposoby leczenia wrodzonej amaurozy-1 Lebera (LCA1) |
| PL18198722T PL3486320T3 (pl) | 2010-04-23 | 2011-04-22 | Kompozycje RAAV-cyklazy guanylanowej i sposoby leczenia wrodzonej amaurozy-1 Lebera (LCA1) |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL18198722T PL3486320T3 (pl) | 2010-04-23 | 2011-04-22 | Kompozycje RAAV-cyklazy guanylanowej i sposoby leczenia wrodzonej amaurozy-1 Lebera (LCA1) |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US9816108B2 (pl) |
| EP (3) | EP3486320B1 (pl) |
| JP (4) | JP5897549B2 (pl) |
| KR (3) | KR20190060881A (pl) |
| CN (2) | CN105821079B (pl) |
| AU (1) | AU2011242527B2 (pl) |
| BR (1) | BR112012026730A8 (pl) |
| CA (1) | CA2796399C (pl) |
| CL (1) | CL2012002969A1 (pl) |
| CO (1) | CO6640236A2 (pl) |
| CY (1) | CY1123008T1 (pl) |
| DK (2) | DK2561067T3 (pl) |
| EA (2) | EA202091105A1 (pl) |
| ES (2) | ES2911185T3 (pl) |
| HR (1) | HRP20190144T1 (pl) |
| HU (1) | HUE041571T2 (pl) |
| IL (2) | IL222651B (pl) |
| LT (1) | LT2561067T (pl) |
| MX (1) | MX351865B (pl) |
| NZ (1) | NZ602897A (pl) |
| PL (2) | PL2561067T3 (pl) |
| PT (2) | PT2561067T (pl) |
| RS (1) | RS58434B1 (pl) |
| SG (4) | SG10201800541SA (pl) |
| SI (1) | SI2561067T1 (pl) |
| SM (1) | SMT201900097T1 (pl) |
| TR (1) | TR201901377T4 (pl) |
| WO (1) | WO2011133933A2 (pl) |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| KR20150020250A (ko) | 2012-06-19 | 2015-02-25 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 당뇨병을 치료하기 위한 조성물 및 방법 |
| FR3008618A1 (fr) * | 2013-07-19 | 2015-01-23 | Univ Paris Curie | Utilisation de composes pour restaurer la reponse a la lumiere des cellules de la retine |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| AU2014305218B2 (en) | 2013-08-05 | 2020-01-16 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| 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 |
| WO2018009814A1 (en) * | 2016-07-08 | 2018-01-11 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving rdh12 |
| KR102606810B1 (ko) * | 2017-05-05 | 2023-11-27 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 오토펄린을 발현시키기 위한 조성물 및 방법 |
| EP3675913A4 (en) | 2017-08-28 | 2021-06-02 | University of Florida Research Foundation, Incorporated | Methods and compositions for treating cone-rod retinal dystrophy |
| KR102704296B1 (ko) * | 2017-11-15 | 2024-09-05 | 더 리젠츠 오브 더 유니버시티 오브 미시간 | Rdh12 암호화 영역을 포함하는 바이러스 벡터 및 망막 이영양증을 치료하는 방법 |
| WO2019155833A1 (ja) * | 2018-02-07 | 2019-08-15 | 学校法人日本医科大学 | 改良型アデノ随伴ウイルスベクター |
| CN112703016B (zh) | 2018-04-27 | 2024-11-22 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| KR20250141845A (ko) * | 2018-07-11 | 2025-09-29 | 티센토 쎄라퓨틱스 인크. | 미토콘드리아 장애의 치료를 위한 sGC 자극제의 용도 |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| EP3914229A4 (en) * | 2019-01-23 | 2022-11-02 | University of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS |
| CA3129422A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| WO2020168111A1 (en) | 2019-02-15 | 2020-08-20 | Exhaura, Ltd. | Dual leucine zipper kinase inhibitors for gene therapy |
| KR20220130093A (ko) | 2019-10-30 | 2022-09-26 | 데시벨 테라퓨틱스, 인크. | 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법 |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| US20230233708A1 (en) * | 2020-04-14 | 2023-07-27 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated delivery and transduction with polyvinyl alcohol |
| WO2021211614A1 (en) | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
| JP2024016295A (ja) * | 2020-11-30 | 2024-02-07 | タカラバイオ株式会社 | 二価の陽イオンを利用した非エンベロープウイルスの製造方法 |
| CN112852976B (zh) * | 2021-03-17 | 2023-10-31 | 湖北省农业科学院畜牧兽医研究所 | 蛋鸡ncs1基因中与后期产蛋性状相关的分子标记及其应用 |
| CN117980489A (zh) * | 2021-07-14 | 2024-05-03 | 梅里特斯英国第二有限公司 | Retgc基因疗法 |
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