JP2022512974A - 生体内crispr超抑制因子による合成免疫調節 - Google Patents
生体内crispr超抑制因子による合成免疫調節 Download PDFInfo
- Publication number
- JP2022512974A JP2022512974A JP2021525082A JP2021525082A JP2022512974A JP 2022512974 A JP2022512974 A JP 2022512974A JP 2021525082 A JP2021525082 A JP 2021525082A JP 2021525082 A JP2021525082 A JP 2021525082A JP 2022512974 A JP2022512974 A JP 2022512974A
- Authority
- JP
- Japan
- Prior art keywords
- myd88
- subject
- crispr
- grna
- ifn
- 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
Images
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
- C07K14/4703—Inhibitors; Suppressors
-
- 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
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding 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
- 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
-
- 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
- C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
-
- 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
- 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
-
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/85—Fusion polypeptide containing an RNA binding domain
-
- 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
- C12N2310/16—Aptamers
-
- 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 [CRISPR]
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862757679P | 2018-11-08 | 2018-11-08 | |
| US62/757,679 | 2018-11-08 | ||
| US201962899584P | 2019-09-12 | 2019-09-12 | |
| US62/899,584 | 2019-09-12 | ||
| PCT/US2019/060285 WO2020097344A1 (en) | 2018-11-08 | 2019-11-07 | Synthetic immunomodulation with a crispr super-repressor in vivo |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022512974A true JP2022512974A (ja) | 2022-02-07 |
| JP2022512974A5 JP2022512974A5 (https=) | 2023-06-12 |
Family
ID=70611555
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021525082A Pending JP2022512974A (ja) | 2018-11-08 | 2019-11-07 | 生体内crispr超抑制因子による合成免疫調節 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11884925B2 (https=) |
| EP (1) | EP3877398A4 (https=) |
| JP (1) | JP2022512974A (https=) |
| KR (1) | KR20210102247A (https=) |
| CN (1) | CN113330023A (https=) |
| AU (1) | AU2019375914A1 (https=) |
| CA (1) | CA3119302A1 (https=) |
| IL (1) | IL283022A (https=) |
| SG (1) | SG11202104802RA (https=) |
| WO (1) | WO2020097344A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11773407B2 (en) | 2017-06-26 | 2023-10-03 | Arizona Board Of Regents On Behalf Of Arizona State University | CRISPR logic circuits for safer and controllable gene therapies |
| US20200123567A1 (en) | 2017-06-26 | 2020-04-23 | Samira Kiani | A universal platform to enhance crispr-based gene editing for in vivo therapies |
| WO2023288306A2 (en) * | 2021-07-16 | 2023-01-19 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Targeting myd88 gene in vitro and in vivo |
| EP4479538A4 (en) * | 2022-02-17 | 2026-01-28 | Syntax Bio Inc | CELL PROGRAMMING SYSTEMS AND ASSOCIATED PROCESSES |
| EP4711456A1 (en) * | 2024-09-11 | 2026-03-18 | GenCC GmbH & Co. KG | Method for the manufacture of individualized crispr/cas complexes and individualized crispr/cas complexes |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014506896A (ja) * | 2011-03-02 | 2014-03-20 | ▲華▼中科技大学同▲済▼医学院附属同▲済▼医院 | アミノチアゾールmyd88特異的阻害剤の薬学的な使用 |
| WO2016049258A2 (en) * | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| WO2016094872A1 (en) * | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| JP2017506232A (ja) * | 2014-02-07 | 2017-03-02 | Sbiバイオテック株式会社 | 腫瘍を治療するための阻害性オリゴヌクレオチド |
| JP2017532001A (ja) * | 2013-12-12 | 2017-11-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物 |
| JP2018519244A (ja) * | 2015-07-08 | 2018-07-19 | リサーチ アンド ビジネス ファウンデーション ソンギュンクァン ユニバーシティ | ピロリジンカルボキサミド誘導体ならびにその調製および使用方法 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US20030148986A1 (en) * | 2001-12-17 | 2003-08-07 | Cedars-Sinai Medical Center | Methods for treating vascular disease by inhibiting myeloid differentiation factor 88 |
| US9228207B2 (en) * | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| TWI906646B (zh) * | 2015-06-18 | 2025-12-01 | 美商博得學院股份有限公司 | 降低脫靶效應的crispr酶突變 |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2017068077A1 (en) | 2015-10-20 | 2017-04-27 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and products for genetic engineering |
| US11439692B2 (en) * | 2017-05-17 | 2022-09-13 | Modalis Therapeutics Corporation | Method of treating diseases associated with MYD88 pathways using CRISPR-GNDM system |
| US11773407B2 (en) * | 2017-06-26 | 2023-10-03 | Arizona Board Of Regents On Behalf Of Arizona State University | CRISPR logic circuits for safer and controllable gene therapies |
-
2019
- 2019-11-07 SG SG11202104802RA patent/SG11202104802RA/en unknown
- 2019-11-07 CN CN201980088195.0A patent/CN113330023A/zh active Pending
- 2019-11-07 EP EP19881214.1A patent/EP3877398A4/en not_active Withdrawn
- 2019-11-07 KR KR1020217017529A patent/KR20210102247A/ko not_active Ceased
- 2019-11-07 AU AU2019375914A patent/AU2019375914A1/en not_active Abandoned
- 2019-11-07 WO PCT/US2019/060285 patent/WO2020097344A1/en not_active Ceased
- 2019-11-07 US US17/292,407 patent/US11884925B2/en active Active
- 2019-11-07 JP JP2021525082A patent/JP2022512974A/ja active Pending
- 2019-11-07 CA CA3119302A patent/CA3119302A1/en active Pending
-
2021
- 2021-05-07 IL IL283022A patent/IL283022A/en unknown
-
2023
- 2023-12-11 US US18/535,658 patent/US20240117364A1/en active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014506896A (ja) * | 2011-03-02 | 2014-03-20 | ▲華▼中科技大学同▲済▼医学院附属同▲済▼医院 | アミノチアゾールmyd88特異的阻害剤の薬学的な使用 |
| JP2017532001A (ja) * | 2013-12-12 | 2017-11-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物 |
| JP2017506232A (ja) * | 2014-02-07 | 2017-03-02 | Sbiバイオテック株式会社 | 腫瘍を治療するための阻害性オリゴヌクレオチド |
| WO2016049258A2 (en) * | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
| WO2016094872A1 (en) * | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| JP2018519244A (ja) * | 2015-07-08 | 2018-07-19 | リサーチ アンド ビジネス ファウンデーション ソンギュンクァン ユニバーシティ | ピロリジンカルボキサミド誘導体ならびにその調製および使用方法 |
Non-Patent Citations (3)
| Title |
|---|
| ACS CHEMICAL BIOLOGY, vol. 13, JPN6023040017, 16 October 2017 (2017-10-16), pages 406 - 416, ISSN: 0005162238 * |
| NATURE METHODS, vol. 15, JPN6023040018, August 2018 (2018-08-01), pages 611 - 616, ISSN: 0005162237 * |
| NATURE REVIEWS MOLECULAR CELL BIOLOGY, vol. 17, JPN6023040016, 2016, pages 5 - 15, ISSN: 0005162239 * |
Also Published As
| Publication number | Publication date |
|---|---|
| SG11202104802RA (en) | 2021-06-29 |
| EP3877398A1 (en) | 2021-09-15 |
| IL283022A (en) | 2021-06-30 |
| CN113330023A (zh) | 2021-08-31 |
| KR20210102247A (ko) | 2021-08-19 |
| EP3877398A4 (en) | 2022-08-17 |
| US11884925B2 (en) | 2024-01-30 |
| WO2020097344A1 (en) | 2020-05-14 |
| US20210380990A1 (en) | 2021-12-09 |
| CA3119302A1 (en) | 2020-05-14 |
| AU2019375914A1 (en) | 2021-06-03 |
| US20240117364A1 (en) | 2024-04-11 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022512974A (ja) | 生体内crispr超抑制因子による合成免疫調節 | |
| US20230250407A1 (en) | Silencing of dux4 by recombinant gene editing complexes | |
| JP2025114562A (ja) | 非組み込みウイルス送達システムおよびその使用の方法 | |
| EP3586868A2 (en) | Liver-specific nucleic acid regulatory elements and methods and use thereof | |
| EP3573618B1 (en) | Compositions and methods for hemoglobin production | |
| US20250099554A1 (en) | Compositions and methods of fas inhibition | |
| AU2021230361A1 (en) | Compositions and methods for the treatment of Metabolic Liver Disorders | |
| KR20230003511A (ko) | 안면견갑상완 근이영양증에 대한 crispr-억제 | |
| US20230287428A1 (en) | Biallelic knockout of sarm1 | |
| WO2020104783A1 (en) | Regulatory nucleic acid sequences | |
| TW202208412A (zh) | 對酪胺酸蛋白激酶樣7(ptk7)特異的經掩蔽的嵌合抗原受體和表現其的免疫細胞 | |
| KR20250039994A (ko) | Angptl3의 이중 대립유전자 녹아웃 | |
| US20250017970A1 (en) | Reduced Expression of Sarm1 For Use In Cell Therapy | |
| Lopes et al. | Gene Editing for ATXN3 Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity | |
| US20260097123A1 (en) | Biallelic Knockout of CIITA | |
| JP2026071272A (ja) | SARM1の両対立k遺伝子ノックアウト | |
| HK40116920A (zh) | Sarm1表达降低用於细胞疗法 | |
| WO2025180419A1 (zh) | 一种脱氨酶变体、包含其的碱基编辑器及其应用 | |
| WO2026019837A1 (en) | Compositions and methods for overcoming t-cell exhaustion | |
| HK40107069A (zh) | 用於治疗高胆固醇血症的组合物和方法 | |
| JP2003146909A (ja) | 治療用ポリペプチドに対する免疫寛容の誘導 | |
| CN121358865A (zh) | 肝脏特异性调控核酸序列 | |
| HK40094039A (zh) | Sarm1的双等位基因敲除 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20221104 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20221104 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20230531 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20230928 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20240423 |