HRP20240806T1 - Metode liječenja reumatoidnog artritisa pomoću rna-vođenog genoma uređivanja hla gena - Google Patents
Metode liječenja reumatoidnog artritisa pomoću rna-vođenog genoma uređivanja hla gena Download PDFInfo
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- HRP20240806T1 HRP20240806T1 HRP20240806TT HRP20240806T HRP20240806T1 HR P20240806 T1 HRP20240806 T1 HR P20240806T1 HR P20240806T T HRP20240806T T HR P20240806TT HR P20240806 T HRP20240806 T HR P20240806T HR P20240806 T1 HRP20240806 T1 HR P20240806T1
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- Prior art keywords
- nucleic acid
- cell
- codon
- guide rna
- drb1
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- 238000000034 method Methods 0.000 title claims 11
- 206010039073 rheumatoid arthritis Diseases 0.000 title claims 3
- 238000010362 genome editing Methods 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 claims 17
- 108091033409 CRISPR Proteins 0.000 claims 14
- 108020004707 nucleic acids Proteins 0.000 claims 14
- 102000039446 nucleic acids Human genes 0.000 claims 14
- 108020004705 Codon Proteins 0.000 claims 11
- 108020005004 Guide RNA Proteins 0.000 claims 11
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 10
- 108700028369 Alleles Proteins 0.000 claims 9
- 108010029657 HLA-DRB1*04:01 antigen Proteins 0.000 claims 8
- 210000004027 cell Anatomy 0.000 claims 7
- 210000000130 stem cell Anatomy 0.000 claims 7
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 claims 4
- 210000000601 blood cell Anatomy 0.000 claims 4
- 235000013922 glutamic acid Nutrition 0.000 claims 4
- 239000004220 glutamic acid Substances 0.000 claims 4
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 3
- 239000004472 Lysine Substances 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 230000002950 deficient Effects 0.000 claims 3
- 230000010354 integration Effects 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 102100040485 HLA class II histocompatibility antigen, DRB1 beta chain Human genes 0.000 claims 1
- 108010039343 HLA-DRB1 Chains Proteins 0.000 claims 1
- 101150018610 HLA-DRB1 gene Proteins 0.000 claims 1
- 241000714192 Human spumaretrovirus Species 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 108091028664 Ribonucleotide Proteins 0.000 claims 1
- 210000003995 blood forming stem cell Anatomy 0.000 claims 1
- 210000002798 bone marrow cell Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 230000006698 induction Effects 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 210000003643 myeloid progenitor cell Anatomy 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 239000002336 ribonucleotide Substances 0.000 claims 1
- 125000002652 ribonucleotide group Chemical group 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 230000026683 transduction Effects 0.000 claims 1
- 238000010361 transduction Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
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- C—CHEMISTRY; METALLURGY
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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
- 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
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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
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
- 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
- C12N15/1138—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 receptors or cell surface proteins
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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
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- 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 [RNase]; Deoxyribonucleases [DNase]
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
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- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0325—Animal model for autoimmune diseases
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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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- 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 [CRISPR]
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- 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
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- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Medicinal Chemistry (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Environmental Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Rheumatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Mycology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762491487P | 2017-04-28 | 2017-04-28 | |
| EP18791113.6A EP3615674B1 (en) | 2017-04-28 | 2018-04-25 | Methods of treating rheumatoid arthritis using rna-guided genome editing of hla gene |
| PCT/US2018/029302 WO2018200635A1 (en) | 2017-04-28 | 2018-04-25 | Methods of treating rheumatoid arthritis using rna-guided genome editing of hla gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20240806T1 true HRP20240806T1 (hr) | 2024-12-06 |
Family
ID=63918677
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20240806TT HRP20240806T1 (hr) | 2017-04-28 | 2018-04-25 | Metode liječenja reumatoidnog artritisa pomoću rna-vođenog genoma uređivanja hla gena |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US11932867B2 (https=) |
| EP (2) | EP3615674B1 (https=) |
| JP (2) | JP7457302B2 (https=) |
| AU (2) | AU2018258061B2 (https=) |
| CA (1) | CA3061614A1 (https=) |
| DK (1) | DK3615674T3 (https=) |
| ES (1) | ES2981611T3 (https=) |
| FI (1) | FI3615674T3 (https=) |
| HR (1) | HRP20240806T1 (https=) |
| HU (1) | HUE069314T2 (https=) |
| LT (1) | LT3615674T (https=) |
| SI (1) | SI3615674T1 (https=) |
| WO (1) | WO2018200635A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| LT3615674T (lt) | 2017-04-28 | 2024-09-25 | The Regents Of The University Of Colorado, A Body Corporate | Reumatoidinio artrito gydymo būdai, naudojant nukreipiančios rnr hla geno redagavimą genome |
| KR20240023035A (ko) | 2021-05-10 | 2024-02-20 | 더 리젠츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 | 자가면역 치료를 위한 hla 조작 방법 및 조성물 |
| WO2025195458A1 (en) * | 2024-03-20 | 2025-09-25 | Nanjing Legend Biotech Co., Ltd. | Methods for producing immune-compatible cells |
Family Cites Families (44)
| Publication number | Priority date | Publication date | Assignee | Title |
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| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| JP4285766B2 (ja) | 1994-03-23 | 2009-06-24 | オハイオ ユニバーシティ | 緻密にした核酸および細胞へのデリバリ |
| US5965787A (en) * | 1995-08-31 | 1999-10-12 | Mayo Foundation For Medical Education And Research | HLA-DRBI peptides with specific binding affinity for HLA-DQ molecules: prevention and treatment of rheumatoid arthritis |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
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| AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
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- 2018-04-25 FI FIEP18791113.6T patent/FI3615674T3/fi active
- 2018-04-25 SI SI201831109T patent/SI3615674T1/sl unknown
- 2018-04-25 CA CA3061614A patent/CA3061614A1/en active Pending
- 2018-04-25 US US16/609,175 patent/US11932867B2/en active Active
- 2018-04-25 WO PCT/US2018/029302 patent/WO2018200635A1/en not_active Ceased
- 2018-04-25 ES ES18791113T patent/ES2981611T3/es active Active
- 2018-04-25 HR HRP20240806TT patent/HRP20240806T1/hr unknown
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| AU2018258061A1 (en) | 2019-11-28 |
| LT3615674T (lt) | 2024-09-25 |
| EP3615674B1 (en) | 2024-03-13 |
| WO2018200635A1 (en) | 2018-11-01 |
| US11932867B2 (en) | 2024-03-19 |
| JP2024075603A (ja) | 2024-06-04 |
| EP3615674A4 (en) | 2021-03-24 |
| JP7457302B2 (ja) | 2024-03-28 |
| FI3615674T3 (fi) | 2024-06-18 |
| AU2025200803A1 (en) | 2025-03-20 |
| AU2018258061B2 (en) | 2024-11-07 |
| HUE069314T2 (hu) | 2025-03-28 |
| CA3061614A1 (en) | 2018-11-01 |
| JP2020517289A (ja) | 2020-06-18 |
| US20240327862A1 (en) | 2024-10-03 |
| US20200199616A1 (en) | 2020-06-25 |
| SI3615674T1 (sl) | 2025-03-31 |
| ES2981611T3 (es) | 2024-10-09 |
| EP3615674A1 (en) | 2020-03-04 |
| DK3615674T3 (da) | 2024-06-17 |
| EP4400175A3 (en) | 2024-09-25 |
| EP4400175A2 (en) | 2024-07-17 |
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