CA2932478A1 - Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing - Google Patents
Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing Download PDFInfo
- Publication number
- CA2932478A1 CA2932478A1 CA2932478A CA2932478A CA2932478A1 CA 2932478 A1 CA2932478 A1 CA 2932478A1 CA 2932478 A CA2932478 A CA 2932478A CA 2932478 A CA2932478 A CA 2932478A CA 2932478 A1 CA2932478 A1 CA 2932478A1
- Authority
- CA
- Canada
- Prior art keywords
- sequence
- cas9
- tracr
- crispr
- target
- 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
- 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
- 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
-
- 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
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- 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
- A61K48/005—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
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- 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
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, 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
- C12N15/63—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
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
- C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
- C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
-
- 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
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- 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
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Bioinformatics & Computational Biology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361915153P | 2013-12-12 | 2013-12-12 | |
| US201361915215P | 2013-12-12 | 2013-12-12 | |
| US201361915145P | 2013-12-12 | 2013-12-12 | |
| US201361915176P | 2013-12-12 | 2013-12-12 | |
| US201361915192P | 2013-12-12 | 2013-12-12 | |
| US201361915107P | 2013-12-12 | 2013-12-12 | |
| US201361915148P | 2013-12-12 | 2013-12-12 | |
| US61/915,153 | 2013-12-12 | ||
| US61/915,145 | 2013-12-12 | ||
| US61/915,148 | 2013-12-12 | ||
| US61/915,176 | 2013-12-12 | ||
| US61/915,215 | 2013-12-12 | ||
| US61/915,107 | 2013-12-12 | ||
| US61/915,192 | 2013-12-12 | ||
| PCT/US2014/070127 WO2015089462A1 (en) | 2013-12-12 | 2014-12-12 | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2932478A1 true CA2932478A1 (en) | 2015-06-18 |
Family
ID=52273585
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2932478A Pending CA2932478A1 (en) | 2013-12-12 | 2014-12-12 | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11407985B2 (https=) |
| EP (2) | EP3079725B1 (https=) |
| JP (3) | JP7103750B2 (https=) |
| KR (2) | KR102805572B1 (https=) |
| CN (3) | CN111206032B (https=) |
| AU (5) | AU2014361781B2 (https=) |
| BR (1) | BR112016013201B1 (https=) |
| CA (1) | CA2932478A1 (https=) |
| DK (1) | DK3079725T3 (https=) |
| ES (1) | ES2765481T3 (https=) |
| IL (3) | IL316141A (https=) |
| MX (2) | MX374529B (https=) |
| SG (1) | SG10201804976YA (https=) |
| WO (1) | WO2015089462A1 (https=) |
| ZA (1) | ZA201603714B (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018051347A1 (en) * | 2016-09-14 | 2018-03-22 | Yeda Research And Development Co. Ltd. | Crisp-seq, an integrated method for massively parallel single cell rna-seq and crispr pooled screens |
| CN109804069A (zh) * | 2016-09-23 | 2019-05-24 | ProQR治疗上市公司Ⅱ | 治疗眼部疾病的反义寡核苷酸 |
Families Citing this family (282)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| MX384291B (es) | 2013-02-20 | 2025-03-14 | Regeneron Pharma | Modificación genética de ratas. |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| WO2015073978A2 (en) | 2013-11-15 | 2015-05-21 | Avellino Lab Usa, Inc. | Methods for multiplex detection of alleles associated with ophthalmic conditions |
| KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US11028388B2 (en) * | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| ES2745769T3 (es) | 2014-03-10 | 2020-03-03 | Editas Medicine Inc | Procedimientos y composiciones relacionados con CRISPR/CAS para tratar la amaurosis congénita de Leber 10 (LCA10) |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| TWI716367B (zh) * | 2014-10-31 | 2021-01-21 | 麻省理工學院 | 用於常間回文重複序列叢集(crispr)之大量平行組合性基因學 |
| KR102531016B1 (ko) | 2014-11-21 | 2023-05-10 | 리제너론 파마슈티칼스 인코포레이티드 | 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물 |
| EP3224381B1 (en) | 2014-11-25 | 2019-09-04 | The Brigham and Women's Hospital, Inc. | Method of identifying a person having a predisposition to or afflicted with a cardiometabolic disease |
| CA2969619A1 (en) | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| EP3234192B1 (en) | 2014-12-19 | 2021-07-14 | The Broad Institute, Inc. | Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing |
| US10196613B2 (en) | 2014-12-19 | 2019-02-05 | Regeneron Pharmaceuticals, Inc. | Stem cells for modeling type 2 diabetes |
| WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
| WO2016108926A1 (en) | 2014-12-30 | 2016-07-07 | The Broad Institute Inc. | Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| KR102888521B1 (ko) | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| CA2981716C (en) | 2015-04-10 | 2022-04-12 | Feldan Bio Inc. | Polypeptide-based shuttle agents for improving the transduction efficiency of polypeptide cargos to the cytosol of target eukaryotic cells, uses thereof, methods and kits relating to same |
| JP2018522249A (ja) | 2015-04-24 | 2018-08-09 | エディタス・メディシン、インコーポレイテッド | Cas9分子/ガイドrna分子複合体の評価 |
| WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| AU2016279077A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | Novel CRISPR enzymes and systems |
| WO2017004261A1 (en) | 2015-06-29 | 2017-01-05 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| CN108350446A (zh) * | 2015-07-02 | 2018-07-31 | 约翰霍普金斯大学 | 基于crispr/cas9的治疗 |
| US10456452B2 (en) | 2015-07-02 | 2019-10-29 | Poseida Therapeutics, Inc. | Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles |
| CN108025074A (zh) * | 2015-07-25 | 2018-05-11 | 哈比卜·弗罗斯特 | 用于为癌症和其他病理状态提供治疗或治愈的系统、装置和方法 |
| EP4512819A3 (en) | 2015-08-12 | 2025-08-06 | The General Hospital Corporation | Compositions and methods that promote hypoxia or the hypoxia response for treatment and prevention of mitochondrial dysfunction and oxidative stress disorders |
| WO2017035416A2 (en) * | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| US10603363B2 (en) * | 2015-09-08 | 2020-03-31 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| FR3042506B1 (fr) * | 2015-10-16 | 2018-11-30 | IFP Energies Nouvelles | Outil genetique de transformation de bacteries clostridium |
| 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 |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| KR101651817B1 (ko) * | 2015-10-28 | 2016-08-29 | 대한민국 | Ngs 라이브러리 제작용 프라이머 세트 및 이를 이용한 ngs 라이브러리 제작방법 및 키트 |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| AU2016355178B9 (en) | 2015-11-19 | 2019-05-30 | Massachusetts Institute Of Technology | Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| WO2017099494A1 (ko) | 2015-12-08 | 2017-06-15 | 기초과학연구원 | Cpf1을 포함하는 유전체 교정용 조성물 및 그 용도 |
| SG10201913266UA (en) * | 2015-12-11 | 2020-02-27 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| WO2017136520A1 (en) * | 2016-02-04 | 2017-08-10 | President And Fellows Of Harvard College | Mitochondrial genome editing and regulation |
| US20190032088A1 (en) * | 2016-02-26 | 2019-01-31 | Cellectis | Micelle based system nuclease encapsulation for in-vivo gene editing |
| WO2017160662A1 (en) * | 2016-03-12 | 2017-09-21 | The Regents Of The University Of California | Biodegradable vectors for efficient rna delivery |
| EP3219799A1 (en) | 2016-03-17 | 2017-09-20 | IMBA-Institut für Molekulare Biotechnologie GmbH | Conditional crispr sgrna expression |
| US12011488B2 (en) | 2016-03-23 | 2024-06-18 | The Regents Of The University Of California | Methods of treating mitochondrial disorders |
| CN109476716A (zh) | 2016-03-23 | 2019-03-15 | 加利福尼亚大学董事会 | 治疗线粒体障碍的方法 |
| US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| WO2017175072A1 (en) * | 2016-04-08 | 2017-10-12 | Feldan Bio Inc. | Peptide shuttle based gene disruption |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| US11773410B2 (en) * | 2016-04-13 | 2023-10-03 | University Of Massachusetts | Repairing compound heterozygous recessive mutations by allele exchange |
| US11213594B2 (en) | 2016-04-29 | 2022-01-04 | Poseida Therapeutics, Inc. | Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids |
| CN113831407B (zh) | 2016-05-20 | 2024-06-11 | 瑞泽恩制药公司 | 用于使用多个引导rna来破坏免疫耐受性的方法 |
| CN109906271A (zh) * | 2016-06-03 | 2019-06-18 | 国家医疗保健研究所 | 编码Cas9核酸酶的核酸的饮食控制的表达及其用途 |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| US11882815B2 (en) * | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| EP4484443A3 (en) * | 2016-06-29 | 2025-03-26 | CRISPR Therapeutics AG | Materials and methods for treatment of friedreich ataxia and other related disorders |
| US12595478B2 (en) | 2016-06-29 | 2026-04-07 | The Broad Institute, Inc. | Crispr-Cas systems having destabilization domain |
| KR20190039703A (ko) * | 2016-07-05 | 2019-04-15 | 더 존스 홉킨스 유니버시티 | Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법 |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Cpf1に基づくゲノム編集の治療適用 |
| EP3492096A4 (en) * | 2016-07-28 | 2020-04-15 | Institute for Basic Science | PHARMACEUTICAL COMPOSITION FOR THE TREATMENT OF EYE DISEASES WITH CAS9 PROTEINS AND GUIDE RNA |
| BR112019001887A2 (pt) | 2016-08-02 | 2019-07-09 | Editas Medicine Inc | composições e métodos para o tratamento de doença associada a cep290 |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018035387A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| EP4485466A3 (en) | 2016-08-17 | 2025-04-02 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| SG11201901306XA (en) * | 2016-08-19 | 2019-03-28 | Toolgen Inc | Artificially engineered angiogenesis regulatory system |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| US12499971B2 (en) | 2016-09-28 | 2025-12-16 | The Broad Institute, Inc. | Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof |
| US12447213B2 (en) | 2016-10-07 | 2025-10-21 | The Broad Institute, Inc. | Modulation of novel immune checkpoint targets |
| CN110291100B (zh) | 2016-10-12 | 2024-06-11 | 费尔丹生物公司 | 用于将多肽负荷从细胞外空间递送至靶真核细胞的胞质溶胶和/或细胞核的合理设计的合成肽穿梭剂,其用途、与其相关的方法和试剂盒 |
| US9982267B2 (en) | 2016-10-12 | 2018-05-29 | Feldan Bio Inc. | Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same |
| US11629170B2 (en) | 2016-10-12 | 2023-04-18 | Feldan Bio Inc. | Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| CN110139654A (zh) * | 2016-11-03 | 2019-08-16 | 优美佳生物技术有限公司 | 用于细胞重编程的方法和组合物 |
| WO2018089386A1 (en) | 2016-11-11 | 2018-05-17 | The Broad Institute, Inc. | Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action |
| KR101917287B1 (ko) | 2016-12-19 | 2018-11-13 | 한국과학기술연구원 | 자가-조립식 리보뉴클레오단백질 나노입자 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3565608A4 (en) * | 2017-01-05 | 2020-12-16 | Rutgers, The State University of New Jersey | TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES |
| JP7215716B2 (ja) * | 2017-01-13 | 2023-01-31 | 学校法人自治医科大学 | 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター |
| WO2018145111A1 (en) * | 2017-02-06 | 2018-08-09 | Children's Medical Center Corporation | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| WO2018147343A1 (en) * | 2017-02-07 | 2018-08-16 | Edigene Corporation | Method of treating diseases associated with elevated kras expression using crispr-gndm system |
| WO2018164457A1 (ko) * | 2017-03-06 | 2018-09-13 | 기초과학연구원 | C2cL 엔도뉴클레아제를 포함하는 유전체 교정용 조성물 및 이를 이용한 유전체 교정 방법 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| JP2020511141A (ja) * | 2017-03-15 | 2020-04-16 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Cas13bオルソログCRISPR酵素及び系 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| WO2018175924A1 (en) | 2017-03-24 | 2018-09-27 | The Broad Institute, Inc. | Methods and compositions for regulating innate lymphoid cell inflammatory responses |
| US11963966B2 (en) | 2017-03-31 | 2024-04-23 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for treating ovarian tumors |
| US12331319B2 (en) | 2017-04-12 | 2025-06-17 | The Broad Institute, Inc. | Respiratory and sweat gland ionocytes |
| US12590288B2 (en) | 2017-04-12 | 2026-03-31 | The Broad Institute, Inc. | Method of treating an inflammatory disease by administering an agent which binds a surface receptor on a tuft cell that induces an ILC class 2 inflammatory response |
| EP3610266A4 (en) | 2017-04-12 | 2021-04-21 | Massachusetts Eye and Ear Infirmary | TUMOR SIGNATURE OF METASTASIS, COMPOSITIONS OF SUBSTANCES AND USES THEREOF |
| WO2018191750A2 (en) | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
| US12208140B2 (en) | 2017-04-21 | 2025-01-28 | The Broad Institute, Inc. | Targeted delivery to beta cells |
| JOP20190257A1 (ar) * | 2017-04-28 | 2019-10-28 | Novartis Ag | مركبات أريل غير متجانسة ثنائية الحلقة مندمجة 6-6 واستخدامها كمثبطات lats |
| US11591601B2 (en) | 2017-05-05 | 2023-02-28 | The Broad Institute, Inc. | Methods for identification and modification of lncRNA associated with target genotypes and phenotypes |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| EP3622092A4 (en) | 2017-05-11 | 2021-06-23 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS OF USE OF CD8 + TUMOR-INFILTRATING LYMPHOCYTE SUBTYPES AND GENE SIGNATURES THEREOF |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| WO2018213726A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| US11866697B2 (en) | 2017-05-18 | 2024-01-09 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| CN118581154A (zh) * | 2017-06-02 | 2024-09-03 | 国家健康与医学研究院 | 用于遗传障碍的基因疗法的基因组编辑手段和结合病毒载体的基因疗法 |
| CA3102054A1 (en) | 2017-06-05 | 2018-12-13 | Fred Hutchinson Cancer Research Center | Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies |
| WO2018232195A1 (en) | 2017-06-14 | 2018-12-20 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| WO2018232356A1 (en) | 2017-06-15 | 2018-12-20 | The Regents Of The University Of California | Targeted non-viral dna insertions |
| JP7123982B2 (ja) * | 2017-06-15 | 2022-08-23 | ツールゲン インコーポレイテッド | 肝臓において目的のタンパク質を発現するためのプラットフォーム |
| KR102761791B1 (ko) | 2017-06-26 | 2025-02-05 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법 |
| US20200123567A1 (en) | 2017-06-26 | 2020-04-23 | Samira Kiani | A universal platform to enhance crispr-based gene editing for in vivo therapies |
| 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 |
| US20210154326A1 (en) * | 2017-06-26 | 2021-05-27 | Arizona Board Of Regents On Behalf Of Arizona State University | CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear |
| KR102007457B1 (ko) * | 2017-06-30 | 2019-08-05 | 동국대학교 산학협력단 | Cas9 단백질, 가이드 RNA 및 양친매성 펩타이드를 포함하는 나노복합체 및 이의 용도 |
| US12049643B2 (en) | 2017-07-14 | 2024-07-30 | The Broad Institute, Inc. | Methods and compositions for modulating cytotoxic lymphocyte activity |
| US12105089B2 (en) | 2017-07-17 | 2024-10-01 | The Broad Institute, Inc. | Cell atlas of the healthy and ulcerative colitis human colon |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| BR102017016440A2 (pt) | 2017-07-31 | 2019-03-19 | Universidade Federal Do Rio Grande Do Sul | Composição para terapia gênica do sistema nervoso central, processo de obtenção e uso da mesma |
| CA3074202A1 (en) * | 2017-08-28 | 2019-03-07 | Wisconsin Alumni Research Foundation | Nanocapsules for delivering ribonucleoproteins |
| EP3675913A4 (en) * | 2017-08-28 | 2021-06-02 | University of Florida Research Foundation, Incorporated | Methods and compositions for treating cone-rod retinal dystrophy |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| AU2018338318B2 (en) * | 2017-09-21 | 2022-12-22 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
| WO2019071054A1 (en) | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| CN110832596B (zh) * | 2017-10-16 | 2021-03-26 | 因美纳有限公司 | 基于深度学习的深度卷积神经网络训练方法 |
| CA3079681A1 (en) | 2017-10-20 | 2019-04-25 | Fred Hutchinson Cancer Research Center | Systems and methods to produce b cells genetically modified to express selected antibodies |
| CN111867635A (zh) * | 2017-10-20 | 2020-10-30 | 法国国家卫生及研究医学协会 | 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 |
| JP7101419B2 (ja) | 2017-10-27 | 2022-07-15 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 内因性t細胞受容体の標的置換 |
| EP3707258A1 (en) * | 2017-11-06 | 2020-09-16 | Editas Medicine, Inc. | Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy |
| GB2568255A (en) * | 2017-11-08 | 2019-05-15 | Evox Therapeutics Ltd | Exosomes comprising RNA therapeutics |
| US12221720B2 (en) | 2017-11-13 | 2025-02-11 | The Broad Institute, Inc. | Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells |
| WO2019094955A1 (en) | 2017-11-13 | 2019-05-16 | The Broad Institute, Inc. | Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas |
| CA3082450A1 (en) * | 2017-11-21 | 2019-05-31 | Crispr Therapeutics Ag | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
| CA3084185A1 (en) * | 2017-12-06 | 2019-06-13 | Generation Bio Co. | Gene editing using a modified closed-ended dna (cedna) |
| WO2019113506A1 (en) | 2017-12-07 | 2019-06-13 | The Broad Institute, Inc. | Methods and compositions for multiplexing single cell and single nuclei sequencing |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| AU2018393050A1 (en) * | 2017-12-21 | 2020-06-18 | Bayer Healthcare Llc | Materials and methods for treatment of Usher Syndrome Type 2A |
| US11994512B2 (en) | 2018-01-04 | 2024-05-28 | Massachusetts Institute Of Technology | Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| CN109507429A (zh) * | 2018-02-14 | 2019-03-22 | 复旦大学 | 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性位点相互作用蛋白的方法 |
| EP3762040A4 (en) | 2018-03-05 | 2022-01-05 | Children's Medical Center Corporation | COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS |
| WO2019173728A1 (en) * | 2018-03-09 | 2019-09-12 | Lee Hye Young | Crispr-nanoparticles and methods of use in brain disorders |
| KR102907245B1 (ko) | 2018-03-14 | 2026-01-05 | 에디타스 메디신, 인코포레이티드 | 혈색소병증 치료를 위한 시스템 및 방법 |
| US20190284553A1 (en) * | 2018-03-15 | 2019-09-19 | KSQ Therapeutics, Inc. | Gene-regulating compositions and methods for improved immunotherapy |
| BR112020019079A2 (pt) | 2018-03-23 | 2020-12-29 | Massachusetts Eye And Ear Infirmary | Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a |
| EP3773520A4 (en) * | 2018-04-09 | 2021-12-22 | Orgenesis Inc. | BIOXOMES AND REDOXOMES PARTICLES, PROCESS AND COMPOSITION |
| WO2019204585A1 (en) | 2018-04-19 | 2019-10-24 | Massachusetts Institute Of Technology | Single-stranded break detection in double-stranded dna |
| KR20210039983A (ko) * | 2018-04-24 | 2021-04-12 | 리간달 인코포레이티드 | 게놈 편집을 위한 방법 및 조성물 |
| HUE059223T2 (hu) | 2018-04-24 | 2022-10-28 | Kws Saat Se & Co Kgaa | Jobb emészthetõségû növények és marker haplotípusok |
| US11957695B2 (en) | 2018-04-26 | 2024-04-16 | The Broad Institute, Inc. | Methods and compositions targeting glucocorticoid signaling for modulating immune responses |
| WO2019210268A2 (en) | 2018-04-27 | 2019-10-31 | The Broad Institute, Inc. | Sequencing-based proteomics |
| JP2021522787A (ja) * | 2018-04-30 | 2021-09-02 | オレゴン ヘルス アンド サイエンス ユニバーシティ | 遺伝子治療の方法 |
| US20190336585A1 (en) * | 2018-05-03 | 2019-11-07 | John Lawrence Mee | Method for sustainable human cognitive enhancement |
| US20210393796A1 (en) * | 2018-05-03 | 2021-12-23 | Cognigenics, Inc. | Methods for sustainable human cognitive enhancement |
| WO2019213660A2 (en) | 2018-05-04 | 2019-11-07 | The Broad Institute, Inc. | Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| KR20250133482A (ko) * | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| CN108588050B (zh) * | 2018-05-14 | 2021-06-25 | 北京艾克伦医疗科技有限公司 | Dna聚合酶以及核酸检测方法和试剂盒 |
| CN108587998B (zh) * | 2018-05-17 | 2021-05-18 | 浙江大学 | 一种外泌体、外泌体的制备方法及其在制备皮肤浅表性肿瘤的药物中的应用 |
| US20190359985A1 (en) * | 2018-05-22 | 2019-11-28 | John Lawrence Mee | Adjustable method for sustainable human cognitive enhancement |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| CN108707627A (zh) * | 2018-05-31 | 2018-10-26 | 深圳市免疫基因治疗研究院 | 一种mld慢病毒载体、慢病毒及其制备方法和应用 |
| WO2019232542A2 (en) | 2018-06-01 | 2019-12-05 | Massachusetts Institute Of Technology | Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients |
| US12036240B2 (en) | 2018-06-14 | 2024-07-16 | The Broad Institute, Inc. | Compositions and methods targeting complement component 3 for inhibiting tumor growth |
| WO2020051223A1 (en) | 2018-09-04 | 2020-03-12 | The Board Of Regents Of The University Of Texas System | Compositions and methods for organ specific delivery of nucleic acids |
| US12357580B2 (en) | 2018-06-19 | 2025-07-15 | The Board Of Regents Of The University Of Texas System | Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids |
| US20190390193A1 (en) * | 2018-06-23 | 2019-12-26 | John Lawrence Mee | Reversible method for sustainable human cognitive enhancement |
| US20220000988A1 (en) * | 2018-06-23 | 2022-01-06 | John Lawrence Mee | Reversible method for sustainable human cognitive enhancement |
| WO2020006036A1 (en) | 2018-06-26 | 2020-01-02 | Massachusetts Institute Of Technology | Crispr effector system based amplification methods, systems, and diagnostics |
| EP3814519A1 (en) | 2018-06-26 | 2021-05-05 | The Broad Institute, Inc. | Crispr/cas and transposase based amplification compositions, systems and methods |
| IL260445B (en) * | 2018-07-05 | 2019-08-29 | Lempo Therapeutics Ltd | Methods and compositions for modulating myeloperoxidase (mpo) expression |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| EP3827076A1 (en) | 2018-07-26 | 2021-06-02 | Uniwersytet Jagiellonski | In vivo delivery system of the genome dna modifying enzymes and the use thereof |
| CN112888461A (zh) * | 2018-07-26 | 2021-06-01 | 株式会社图尔金 | 抗凝因子的基因编辑 |
| CA3108281A1 (en) * | 2018-08-03 | 2020-02-06 | Beam Therapeutics Inc. | Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence |
| JP2021532815A (ja) | 2018-08-07 | 2021-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Cas12b酵素およびシステム |
| US20210324357A1 (en) | 2018-08-20 | 2021-10-21 | The Brigham And Women's Hospital, Inc. | Degradation domain modifications for spatio-temporal control of rna-guided nucleases |
| US12421507B2 (en) | 2018-08-20 | 2025-09-23 | The Broad Institute, Inc. | Methods and compositions for optochemical control of CRISPR-CAS9 |
| EP3846857A4 (en) * | 2018-09-04 | 2022-10-12 | The Board Of Regents Of The University Of Texas System | COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS |
| CN109402115B (zh) * | 2018-09-06 | 2024-02-02 | 广州普世利华科技有限公司 | 靶向Rett突变基因RNA的gRNA及Rett突变基因的检测方法、检测试剂盒 |
| WO2020051507A1 (en) | 2018-09-06 | 2020-03-12 | The Broad Institute, Inc. | Nucleic acid assemblies for use in targeted delivery |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| WO2020056170A1 (en) | 2018-09-12 | 2020-03-19 | Fred Hutchinson Cancer Research Center | Reducing cd33 expression to selectively protect therapeutic cells |
| AU2019343045A1 (en) | 2018-09-18 | 2021-05-13 | Vnv Newco Inc. | ARC-based capsids and uses thereof |
| US20210355492A1 (en) * | 2018-09-21 | 2021-11-18 | City University Of Hong Kong | Cargo loaded extracellular vesicles |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| US20220411783A1 (en) | 2018-10-12 | 2022-12-29 | The Broad Institute, Inc. | Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues |
| US20210379057A1 (en) | 2018-10-16 | 2021-12-09 | Massachusetts Institute Of Technology | Nutlin-3a for use in treating a mycobacterium tuberculosis infection |
| CN109957568B (zh) * | 2018-10-16 | 2024-02-02 | 广州普世利华科技有限公司 | 用于靶向HBB RNA的gRNA及基于C2c2的HBB突变检测方法、检测试剂盒 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020097370A2 (en) | 2018-11-08 | 2020-05-14 | Triton Algae Innovations, Inc. | Methods for overproducing protoporphyrin ix in algae and compositions therefrom |
| US12402610B2 (en) | 2018-11-09 | 2025-09-02 | The Broad Institute, Inc. | Methods and compositions for modulating innate lymphoid cell pathogenic effectors |
| US12165743B2 (en) | 2018-11-09 | 2024-12-10 | The Broad Institute, Inc. | Compressed sensing for screening and tissue imaging |
| AU2019396520A1 (en) * | 2018-12-12 | 2021-05-27 | Flagship Pioneering Innovations V, Inc. | Anellosomes for delivering secreted therapeutic modalities |
| US20220028491A1 (en) * | 2018-12-13 | 2022-01-27 | The General Hospital Corporation | Biologically informed and accurate sequence alignment |
| CN111321171A (zh) * | 2018-12-14 | 2020-06-23 | 江苏集萃药康生物科技有限公司 | 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法 |
| US20220062394A1 (en) | 2018-12-17 | 2022-03-03 | The Broad Institute, Inc. | Methods for identifying neoantigens |
| CN113544266A (zh) | 2018-12-17 | 2021-10-22 | 博德研究所 | Crispr相关转座酶系统和其使用方法 |
| EP3897745A1 (en) * | 2018-12-23 | 2021-10-27 | CSL Behring LLC | Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome |
| KR102386498B1 (ko) * | 2018-12-27 | 2022-04-15 | 한양대학교 산학협력단 | Crispr-cas를 기반으로 하는 유전자 교정용 조성물 |
| WO2020139031A1 (ko) * | 2018-12-27 | 2020-07-02 | 한양대학교 산학협력단 | Crispr-cas를 기반으로 하는 유전자 교정용 조성물 |
| US11739156B2 (en) | 2019-01-06 | 2023-08-29 | The Broad Institute, Inc. Massachusetts Institute of Technology | Methods and compositions for overcoming immunosuppression |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| CA3130515A1 (en) * | 2019-02-25 | 2020-09-03 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| US20220047618A1 (en) * | 2019-02-28 | 2022-02-17 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| WO2020181193A1 (en) * | 2019-03-06 | 2020-09-10 | The Broad Institute, Inc. | T:a to a:t base editing through adenosine methylation |
| US12534714B2 (en) | 2019-03-18 | 2026-01-27 | The Broad Institute, Inc. | Type VII CRISPR proteins and systems |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| SG11202110613SA (en) * | 2019-03-27 | 2021-10-28 | Res Inst Nationwide Childrens Hospital | Generation of chimeric antigen receptor (car)-primary nk cells for cancer immunotherapy using a combination of cas9/rnp and aav viruses |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3040645A1 (en) | 2019-04-18 | 2020-10-18 | Feldan Bio Inc. | Peptide-based non-proteinaceous cargo delivery |
| CN110129368B (zh) * | 2019-04-22 | 2021-03-09 | 中国科学院脑科学与智能技术卓越创新中心 | 一种感染支持细胞和毛细胞的aav载体 |
| WO2020229533A1 (en) | 2019-05-13 | 2020-11-19 | KWS SAAT SE & Co. KGaA | Drought tolerance in corn |
| WO2020236972A2 (en) | 2019-05-20 | 2020-11-26 | The Broad Institute, Inc. | Non-class i multi-component nucleic acid targeting systems |
| AR118995A1 (es) | 2019-05-25 | 2021-11-17 | Kws Saat Se & Co Kgaa | Mejorador de la inducción de haploides |
| WO2020243661A1 (en) | 2019-05-31 | 2020-12-03 | The Broad Institute, Inc. | Methods for treating metabolic disorders by targeting adcy5 |
| CN110241098B (zh) * | 2019-06-05 | 2021-04-30 | 复旦大学 | 酿脓链球菌的CRISPR核酸酶SpCas9的截短型高特异性变异体及其应用 |
| WO2020252455A1 (en) | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| CN112143701A (zh) * | 2019-06-26 | 2020-12-29 | 中国科学院脑科学与智能技术卓越创新中心 | 基于rna定点编辑的抑制脉络膜新生血管形成的方法及试剂 |
| SG11202111943UA (en) | 2019-07-02 | 2021-11-29 | Hutchinson Fred Cancer Res | Recombinant ad35 vectors and related gene therapy improvements |
| US20230033299A1 (en) * | 2019-07-30 | 2023-02-02 | NxGen Medicine Inc. | Methods of Treating Pain Conditions and Compositions Related Thereto |
| EP3772542A1 (en) | 2019-08-07 | 2021-02-10 | KWS SAAT SE & Co. KGaA | Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2 |
| EP4013868A4 (en) * | 2019-08-16 | 2023-08-23 | The Jackson Laboratory | LIVE CELL IMAGING OF NON-REPETITIVE GENOMIC LOCI |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| US11981922B2 (en) | 2019-10-03 | 2024-05-14 | Dana-Farber Cancer Institute, Inc. | Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| EP4045522A1 (en) | 2019-10-17 | 2022-08-24 | KWS SAAT SE & Co. KGaA | Enhanced disease resistance of crops by downregulation of repressor genes |
| US12521451B2 (en) * | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| CN111001044A (zh) * | 2019-12-30 | 2020-04-14 | 上海申淇医疗科技有限公司 | 一种药物球囊、其涂覆药物的制备及药物球囊的制备方法 |
| KR102152189B1 (ko) | 2020-02-11 | 2020-09-04 | 재단법인 아산사회복지재단 | T 세포 내 표적 위치로의 t 세포 수용체 삽입을 위한 재조합벡터 및 이를 이용한 t 세포 내 표적 위치로의 t 세포 수용체 삽입용 조성물 |
| EP3872190A1 (en) | 2020-02-26 | 2021-09-01 | Antibodies-Online GmbH | A method of using cut&run or cut&tag to validate crispr-cas targeting |
| AU2021257213A1 (en) * | 2020-04-17 | 2022-11-03 | Nevada Research & Innovation Corporation | CRISPR-inhibition for facioscapulohumeral muscular dystrophy |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| GB202007199D0 (en) * | 2020-05-15 | 2020-07-01 | Oxford Biomedica Ltd | Viral vector production |
| AR122206A1 (es) | 2020-05-29 | 2022-08-24 | Kws Saat Se & Co Kgaa | Inducción de haploides en plantas |
| CA3189601A1 (en) | 2020-07-24 | 2022-01-27 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| CN114250228A (zh) * | 2020-09-22 | 2022-03-29 | 广州瑞风生物科技有限公司 | 靶向CTGF基因的gRNA及其应用 |
| WO2022067034A1 (en) * | 2020-09-24 | 2022-03-31 | Albert Einstein College Of Medicine | Attb cell line, transgenic cell lines derived therefrom, and methods of making the same |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| EP4001429A1 (en) | 2020-11-16 | 2022-05-25 | Antibodies-Online GmbH | Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq) |
| CN113057969A (zh) * | 2021-03-09 | 2021-07-02 | 吴志新 | 一种间充质干细胞对儿童自闭症临床应用实验方法 |
| CN113181376B (zh) * | 2021-03-17 | 2022-11-29 | 复旦大学附属眼耳鼻喉科医院 | Htra2基因表达抑制剂在预防获得性感音神经性聋中的应用 |
| CN113667734B (zh) * | 2021-07-16 | 2022-05-24 | 四川大学华西医院 | Shank3片段序列甲基化检测试剂在制备精神分裂症诊断试剂盒中的用途 |
| WO2023006933A1 (en) | 2021-07-30 | 2023-02-02 | KWS SAAT SE & Co. KGaA | Plants with improved digestibility and marker haplotypes |
| JP2024534945A (ja) | 2021-09-10 | 2024-09-26 | アジレント・テクノロジーズ・インク | 化学修飾を有するプライム編集のためのガイドrna |
| JP2024537991A (ja) | 2021-10-14 | 2024-10-18 | アーセナル バイオサイエンシズ インコーポレイテッド | 共発現されるshRNAと論理ゲートシステムとを有する免疫細胞 |
| EP4441232A2 (en) * | 2021-12-01 | 2024-10-09 | Shape Therapeutics Inc. | Functional aav capsids for intravitreal administration |
| WO2023143609A1 (en) * | 2022-01-30 | 2023-08-03 | Edigene Therapeutics (Beijing) Inc. | Methods for nucleic acid editing to alter apoe4 function |
| CN115487315B (zh) * | 2022-04-20 | 2023-08-04 | 暨南大学 | 治疗亨廷顿病的药物 |
| CN115029346B (zh) * | 2022-04-22 | 2024-01-02 | 复旦大学附属眼耳鼻喉科医院 | 用于靶向敲降Htra2转录本的sgRNA、CRISPR/CasRx系统及应用 |
| CA3265393A1 (en) | 2022-08-26 | 2024-02-29 | KWS SAAT SE & Co. KGaA | USE OF PAIRED GENES FOR HYBRID SELECTION |
| WO2024112036A1 (ko) * | 2022-11-21 | 2024-05-30 | 고려대학교 산학협력단 | Cas9 단백질을 이용한 고효율 어댑터 연결 반응에서 어댑터 이합체의 제거 방법 |
| CN116515908B (zh) * | 2023-04-03 | 2024-04-26 | 苏州启辰生物科技有限公司 | Rpe65基因编辑模型犬的建立方法 |
| WO2024236547A1 (en) | 2023-05-18 | 2024-11-21 | Inceptor Bio, Llc | Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof |
| WO2024253957A1 (en) | 2023-06-07 | 2024-12-12 | Northwestern University | Targeting autoimmunity-associated t cells through regulation of transcription factors |
| CN121729237A (zh) * | 2023-06-30 | 2026-03-24 | 武田药品工业株式会社 | Htt阻遏物及其用途 |
| WO2025027136A1 (en) | 2023-08-01 | 2025-02-06 | ETH Zürich | Single-cell crispr-screening of multiple gene perturbations in vivo |
| WO2025046513A1 (en) | 2023-08-29 | 2025-03-06 | Inceptor Bio, Llc | Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof |
| WO2025184403A2 (en) * | 2024-02-29 | 2025-09-04 | Memorial Sloan-Kettering Cancer Center | Cell penetrating peptides and methods including the same |
| WO2025196502A1 (en) | 2024-03-20 | 2025-09-25 | North Carolina Agricultural & Technical State University | Choline kinase inhibitors as a therapeutic treatment for obesity |
| US20260022380A1 (en) | 2024-06-10 | 2026-01-22 | Northwestern University | Genetic modulators of kras protein expression and their uses |
| WO2026047626A1 (en) | 2024-08-30 | 2026-03-05 | Inceptor Bio, Llc | Compositions and methods involving immune cells engineered for binding bispecific antibodies for use in cell therapy |
| WO2026060399A2 (en) | 2024-09-16 | 2026-03-19 | Northwestern University | T cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies |
| WO2026060398A2 (en) | 2024-09-16 | 2026-03-19 | Northwestern University | B cell lymphoma mutations, compositions, and uses to enhance engineered t cell therapies |
| CN119745798B (zh) * | 2024-11-07 | 2025-10-31 | 河北中琨生物工程有限责任公司 | 搭载siCCR5的工程化脐带干细胞外泌体、制备方法及其在治疗阿尔茨海默症上的应用 |
Family Cites Families (237)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| DE122007000007I2 (de) | 1986-04-09 | 2010-12-30 | Genzyme Corp | Genetisch transformierte Tiere, die ein gewünschtes Protein in Milch absondern |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US4873316A (en) | 1987-06-23 | 1989-10-10 | Biogen, Inc. | Isolation of exogenous recombinant proteins from the milk of transgenic mammals |
| US5703055A (en) | 1989-03-21 | 1997-12-30 | Wisconsin Alumni Research Foundation | Generation of antibodies through lipid mediated DNA delivery |
| 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 |
| US7150982B2 (en) | 1991-09-09 | 2006-12-19 | Third Wave Technologies, Inc. | RNA detection assays |
| 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 |
| US5593972A (en) | 1993-01-26 | 1997-01-14 | The Wistar Institute | Genetic immunization |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US6007845A (en) | 1994-07-22 | 1999-12-28 | Massachusetts Institute Of Technology | Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers |
| US7745416B2 (en) | 1995-04-11 | 2010-06-29 | The Regents Of The University Of California | Method for in vivo regulation of cardiac muscle contractility |
| AU698739B2 (en) | 1995-06-06 | 1998-11-05 | Isis Pharmaceuticals, Inc. | Oligonucleotides having phosphorothioate linkages of high chiral purity |
| US5985662A (en) | 1995-07-13 | 1999-11-16 | Isis Pharmaceuticals Inc. | Antisense inhibition of hepatitis B virus replication |
| US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
| US5855913A (en) | 1997-01-16 | 1999-01-05 | Massachusetts Instite Of Technology | Particles incorporating surfactants for pulmonary drug delivery |
| US5985309A (en) | 1996-05-24 | 1999-11-16 | Massachusetts Institute Of Technology | Preparation of particles for inhalation |
| US5846946A (en) | 1996-06-14 | 1998-12-08 | Pasteur Merieux Serums Et Vaccins | Compositions and methods for administering Borrelia DNA |
| GB9907461D0 (en) | 1999-03-31 | 1999-05-26 | King S College London | Neurite regeneration |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| GB9720465D0 (en) | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
| EP1025217B1 (en) | 1997-10-24 | 2006-10-04 | Invitrogen Corporation | Recombinational cloning using nucleic acids having recombination sites |
| US6750059B1 (en) | 1998-07-16 | 2004-06-15 | Whatman, Inc. | Archiving of vectors |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| GB0024550D0 (https=) | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| US7033744B2 (en) | 2001-03-16 | 2006-04-25 | Naoya Kobayashi | Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof |
| EP1385538B1 (en) | 2001-04-05 | 2013-01-30 | The Johns Hopkins University | Chimeric Vaccines comprising the lumenal domain of LAMP-1 or LAMP-2 |
| IL159756A0 (en) | 2001-07-12 | 2004-06-20 | Univ Massachusetts | IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING |
| EP1572885A2 (en) | 2001-08-08 | 2005-09-14 | Genzyme Corporation | Methods for treating diabetes and other blood sugar disorders |
| WO2003016338A1 (en) | 2001-08-15 | 2003-02-27 | Parker Hughes Institute | Crystal structure of the btk kinase domain |
| US7776321B2 (en) | 2001-09-26 | 2010-08-17 | Mayo Foundation For Medical Education And Research | Mutable vaccines |
| GB0125216D0 (en) | 2001-10-19 | 2001-12-12 | Univ Strathclyde | Dendrimers for use in targeted delivery |
| CN1620508A (zh) | 2001-12-21 | 2005-05-25 | 牛津生物医学(英国)有限公司 | 转基因生物 |
| US7206639B2 (en) | 2002-03-15 | 2007-04-17 | Sarcos Investments Lc | Cochlear drug delivery system and method |
| US7539579B2 (en) | 2002-04-09 | 2009-05-26 | Beattie Kenneth L | Oligonucleotide probes for genosensor chips |
| JP2006513694A (ja) | 2002-06-11 | 2006-04-27 | ザ スクリップス リサーチ インスティテュート | 人工転写因子 |
| EP1519714B1 (en) | 2002-06-28 | 2010-10-20 | Protiva Biotherapeutics Inc. | Method and apparatus for producing liposomes |
| GB0220467D0 (en) | 2002-09-03 | 2002-10-09 | Oxford Biomedica Ltd | Composition |
| CA2499188A1 (en) | 2002-09-27 | 2004-04-08 | Cold Spring Harbor Laboratory | Cell-based rna interference and related methods and compositions |
| WO2004046321A2 (en) | 2002-11-15 | 2004-06-03 | Trustees Of Boston University | Cis/trans riboregulators |
| US20060178297A1 (en) | 2003-01-28 | 2006-08-10 | Troy Carol M | Systems and methods for silencing expression of a gene in a cell and uses thereof |
| NZ545000A (en) | 2003-07-03 | 2008-08-29 | Univ California | Genome mapping of functional DNA elements and cellular proteins |
| CA2532228C (en) | 2003-07-16 | 2017-02-14 | Protiva Biotherapeutics, Inc. | Lipid encapsulated interfering rna |
| US20070134796A1 (en) | 2005-07-26 | 2007-06-14 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| AU2004263865B2 (en) | 2003-08-08 | 2007-05-17 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| EP1664316B1 (en) | 2003-09-15 | 2012-08-29 | Protiva Biotherapeutics Inc. | Polyethyleneglycol-modified lipid compounds and uses thereof |
| GB0325379D0 (en) | 2003-10-30 | 2003-12-03 | Oxford Biomedica Ltd | Vectors |
| US8673634B2 (en) | 2003-11-13 | 2014-03-18 | Massachusetts Eye & Ear Infirmary | Method for the treatment of hearing loss |
| FR2862659B1 (fr) | 2003-11-21 | 2006-02-10 | Pasteur Institut | Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques |
| US8053232B2 (en) | 2004-01-23 | 2011-11-08 | Virxsys Corporation | Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing |
| US20070244031A1 (en) | 2004-01-27 | 2007-10-18 | Quan Lu | Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts |
| WO2005072793A1 (en) | 2004-01-29 | 2005-08-11 | The Charles Stark Draper Laboratory, Inc. | Implantable drug delivery apparatus |
| US20050220796A1 (en) | 2004-03-31 | 2005-10-06 | Dynan William S | Compositions and methods for modulating DNA repair |
| CA2569664C (en) | 2004-06-07 | 2013-07-16 | Protiva Biotherapeutics, Inc. | Lipid encapsulated interfering rna |
| JP4764426B2 (ja) | 2004-06-07 | 2011-09-07 | プロチバ バイオセラピューティクス インコーポレイティッド | カチオン性脂質および使用方法 |
| FR2872170B1 (fr) | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| AU2005274948B2 (en) | 2004-07-16 | 2011-09-22 | Genvec, Inc. | Vaccines against aids comprising CMV/R-nucleic acid constructs |
| GB0422877D0 (en) | 2004-10-14 | 2004-11-17 | Univ Glasgow | Bioactive polymers |
| WO2006116756A1 (en) | 2005-04-28 | 2006-11-02 | Benitec, Limited. | Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns |
| US7892224B2 (en) | 2005-06-01 | 2011-02-22 | Brainlab Ag | Inverse catheter planning |
| DK2341149T3 (en) | 2005-08-26 | 2017-02-27 | Dupont Nutrition Biosci Aps | Use of CRISPR-associated genes (Cas) |
| US9265933B2 (en) | 2005-09-08 | 2016-02-23 | Massachusetts Eye And Ear Infirmary | Cochlear implants containing biological cells and uses thereof |
| WO2007048046A2 (en) | 2005-10-20 | 2007-04-26 | Protiva Biotherapeutics, Inc. | Sirna silencing of filovirus gene expression |
| US8044019B2 (en) | 2005-10-28 | 2011-10-25 | Mitsubishi Tanabe Pharma Corporation | Cell penetrating peptide |
| CN101346393B (zh) | 2005-11-02 | 2015-07-22 | 普洛体维生物治疗公司 | 修饰的siRNA分子及其应用 |
| GB0526211D0 (en) | 2005-12-22 | 2006-02-01 | Oxford Biomedica Ltd | Viral vectors |
| EP1989307B1 (en) | 2006-02-08 | 2012-08-08 | Quark Pharmaceuticals, Inc. | NOVEL TANDEM siRNAS |
| WO2007093836A1 (en) | 2006-02-13 | 2007-08-23 | Cellectis | Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof |
| US9677123B2 (en) | 2006-03-15 | 2017-06-13 | Siemens Healthcare Diagnostics Inc. | Degenerate nucleobase analogs |
| CN101484588B (zh) | 2006-05-11 | 2013-11-06 | 阿尔尼拉姆医药品有限公司 | 抑制pcsk9基因表达的组合物和方法 |
| WO2008054544A2 (en) | 2006-05-22 | 2008-05-08 | Immune Disease Institute, Inc. | Method for delivery across the blood brain barrier |
| US7915399B2 (en) | 2006-06-09 | 2011-03-29 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| WO2008010009A1 (en) | 2006-07-18 | 2008-01-24 | Cellectis | Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof |
| JP2008078613A (ja) | 2006-08-24 | 2008-04-03 | Rohm Co Ltd | 窒化物半導体の製造方法及び窒化物半導体素子 |
| US20100146651A1 (en) | 2006-11-14 | 2010-06-10 | Cellectis | Meganuclease variants cleaving a dna target sequence from the hprt gene and uses thereof |
| WO2008076556A2 (en) * | 2006-11-15 | 2008-06-26 | Massachusetts Eye & Ear Infirmary | Generation of inner ear cells |
| WO2008093152A1 (en) | 2007-02-01 | 2008-08-07 | Cellectis | Obligate heterodimer meganucleases and uses thereof |
| CN101688241B (zh) | 2007-03-02 | 2015-01-21 | 杜邦营养生物科学有限公司 | 具有改善的噬菌体抗性的培养物 |
| PE20090064A1 (es) | 2007-03-26 | 2009-03-02 | Novartis Ag | Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende |
| WO2009013559A1 (en) | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| WO2009019528A1 (en) | 2007-08-03 | 2009-02-12 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof |
| SG183726A1 (en) | 2007-08-14 | 2012-09-27 | Hutchinson Fred Cancer Res | Needle array assembly and method for delivering therapeutic agents |
| US8404658B2 (en) | 2007-12-31 | 2013-03-26 | Nanocor Therapeutics, Inc. | RNA interference for the treatment of heart failure |
| US8188131B2 (en) | 2008-02-07 | 2012-05-29 | Massachusetts Eye & Ear Infirmary | Compounds that enhance Atoh1 expression |
| US20100081707A1 (en) | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for nucleic acid delivery |
| WO2009147368A1 (en) | 2008-06-04 | 2009-12-10 | Medical Research Council | Peptides |
| WO2009147684A2 (en) | 2008-06-06 | 2009-12-10 | Quark Pharmaceuticals, Inc. | Compositions and methods for treatment of ear disorders |
| BRPI0910198A2 (pt) | 2008-06-30 | 2016-01-12 | Silenseed Ltd | composição para administração a partir de um sítio local de um agente baseado em nucleotídeos e método para tratar um sujeito sofrendo de uma doença requerendo tratamento local com um agente baseado em nucleotídeo. |
| US20110117189A1 (en) | 2008-07-08 | 2011-05-19 | S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. | Ophthalmic compositions for treating pathologies of the posterior segment of the eye |
| US8496957B2 (en) * | 2008-07-21 | 2013-07-30 | Otonomy, Inc | Controlled release auris sensory cell modulator compositions and methods for the treatment of otic disorders |
| WO2010011961A2 (en) | 2008-07-25 | 2010-01-28 | University Of Georgia Research Foundation, Inc. | Prokaryotic rnai-like system and methods of use |
| WO2010014218A2 (en) | 2008-07-29 | 2010-02-04 | Academia Sinica | Puf-a and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders |
| CN102177235A (zh) | 2008-09-08 | 2011-09-07 | 赛莱克蒂斯公司 | 切割来自谷氨酰胺合成酶基因的dna靶序列的大范围核酸酶变体及其用途 |
| US8383604B2 (en) | 2008-09-15 | 2013-02-26 | Children's Medical Center Corporation | Modulation of BCL11A for treatment of hemoglobinopathies |
| US20100076057A1 (en) | 2008-09-23 | 2010-03-25 | Northwestern University | TARGET DNA INTERFERENCE WITH crRNA |
| JP5756016B2 (ja) | 2008-10-29 | 2015-07-29 | サンガモ バイオサイエンシーズ, インコーポレイテッド | グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物 |
| US9404098B2 (en) | 2008-11-06 | 2016-08-02 | University Of Georgia Research Foundation, Inc. | Method for cleaving a target RNA using a Cas6 polypeptide |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| US20110023145A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in autism spectrum disorders |
| US20120159653A1 (en) | 2008-12-04 | 2012-06-21 | Sigma-Aldrich Co. | Genomic editing of genes involved in macular degeneration |
| US20110016540A1 (en) | 2008-12-04 | 2011-01-20 | Sigma-Aldrich Co. | Genome editing of genes associated with trinucleotide repeat expansion disorders in animals |
| US20110023153A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in alzheimer's disease |
| US20110023144A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in amyotrophyic lateral sclerosis disease |
| US20110023139A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in cardiovascular disease |
| US20110023146A1 (en) | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in secretase-associated disorders |
| CA2746514C (en) | 2008-12-10 | 2018-11-27 | Alnylam Pharmaceuticals, Inc. | Gnaq targeted dsrna compositions and methods for inhibiting expression |
| WO2010075424A2 (en) | 2008-12-22 | 2010-07-01 | The Regents Of University Of California | Compositions and methods for downregulating prokaryotic genes |
| US20110239315A1 (en) | 2009-01-12 | 2011-09-29 | Ulla Bonas | Modular dna-binding domains and methods of use |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| EP2408921B1 (en) | 2009-03-20 | 2017-04-19 | Sangamo BioSciences, Inc. | Modification of cxcr4 using engineered zinc finger proteins |
| TWI576432B (zh) | 2009-04-07 | 2017-04-01 | 陶氏農業科學公司 | 序列特異性核酸酶之奈米粒子媒介式遞送技術 |
| US20120164118A1 (en) | 2009-05-04 | 2012-06-28 | Fred Hutchinson Cancer Research Center | Cocal vesiculovirus envelope pseudotyped retroviral vectors |
| WO2011036510A1 (en) | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof |
| KR20100133319A (ko) | 2009-06-11 | 2010-12-21 | 주식회사 툴젠 | 표적 특이적인 게놈의 재배열을 위한 표적 특이적 뉴클레아제 및 이의 용도 |
| WO2011000107A1 (en) | 2009-07-01 | 2011-01-06 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for delivery of therapeutic agents to solid tumors |
| EP2449106B1 (en) | 2009-07-01 | 2015-04-08 | Protiva Biotherapeutics Inc. | Compositions and methods for silencing apolipoprotein b |
| EP2456877A4 (en) | 2009-07-24 | 2012-05-30 | Sigma Aldrich Co Llc | METHOD OF GENOMIC PROCESSING |
| CA2769262C (en) | 2009-07-28 | 2019-04-30 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
| US8927807B2 (en) | 2009-09-03 | 2015-01-06 | The Regents Of The University Of California | Nitrate-responsive promoter |
| US8889394B2 (en) | 2009-09-07 | 2014-11-18 | Empire Technology Development Llc | Multiple domain proteins |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| EP2504439B1 (en) | 2009-11-27 | 2016-03-02 | BASF Plant Science Company GmbH | Optimized endonucleases and uses thereof |
| WO2011072246A2 (en) | 2009-12-10 | 2011-06-16 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| WO2011076873A1 (en) | 2009-12-23 | 2011-06-30 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Influenza targets |
| EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US10087431B2 (en) | 2010-03-10 | 2018-10-02 | The Regents Of The University Of California | Methods of generating nucleic acid fragments |
| US8927514B2 (en) * | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| MX2012013037A (es) | 2010-05-10 | 2013-07-29 | Univ California | Composiciones de endorribonucleasa y metodos de uso de las mismas. |
| US20130183282A1 (en) | 2010-05-12 | 2013-07-18 | Cellectis | Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof |
| US8372951B2 (en) | 2010-05-14 | 2013-02-12 | National Tsing Hua University | Cell penetrating peptides for intracellular delivery |
| WO2011148194A1 (en) | 2010-05-28 | 2011-12-01 | Oxford Biomedica (Uk) Ltd | Delivery of lentiviral vectors to the brain |
| US9512444B2 (en) | 2010-07-23 | 2016-12-06 | Sigma-Aldrich Co. Llc | Genome editing using targeting endonucleases and single-stranded nucleic acids |
| DK2601611T3 (da) | 2010-08-02 | 2021-02-01 | Integrated Dna Tech Inc | Fremgangsmåder til forudsigelse af stabilitet og smeltetemperaturer for nukleinsyreduplekser |
| US9193827B2 (en) | 2010-08-26 | 2015-11-24 | Massachusetts Institute Of Technology | Poly(beta-amino alcohols), their preparation, and uses thereof |
| US9549901B2 (en) | 2010-09-03 | 2017-01-24 | The Brigham And Women's Hospital, Inc. | Lipid-polymer hybrid particles |
| US9175280B2 (en) | 2010-10-12 | 2015-11-03 | Sangamo Biosciences, Inc. | Methods and compositions for treating hemophilia B |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| US20120204282A1 (en) | 2011-02-04 | 2012-08-09 | Sangamo Biosciences, Inc. | Methods and compositions for treating occular disorders |
| EP2691443B1 (en) | 2011-03-28 | 2021-02-17 | Massachusetts Institute of Technology | Conjugated lipomers and uses thereof |
| AU2012236099A1 (en) | 2011-03-31 | 2013-10-03 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| CN105755044A (zh) * | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| AU2012249390B2 (en) | 2011-04-27 | 2017-03-30 | Amyris, Inc. | Methods for genomic modification |
| US20120295960A1 (en) | 2011-05-20 | 2012-11-22 | Oxford Biomedica (Uk) Ltd. | Treatment regimen for parkinson's disease |
| WO2012164565A1 (en) | 2011-06-01 | 2012-12-06 | Yeda Research And Development Co. Ltd. | Compositions and methods for downregulating prokaryotic genes |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| JP2014530603A (ja) | 2011-10-06 | 2014-11-20 | サンガモ バイオサイエンシーズ, インコーポレイテッド | Hiv感染を制御するための方法および組成物 |
| CA3099582A1 (en) | 2011-10-27 | 2013-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| US20130122096A1 (en) | 2011-11-14 | 2013-05-16 | Silenseed Ltd. | Compositions for drug delivery and methods of manufacturing and using same |
| EP2780376B1 (en) | 2011-11-18 | 2017-03-08 | Université Laval | Methods and products for increasing frataxin levels and uses thereof |
| HK1202434A1 (en) | 2011-11-22 | 2015-10-02 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| US8450107B1 (en) | 2011-11-30 | 2013-05-28 | The Broad Institute Inc. | Nucleotide-specific recognition sequences for designer TAL effectors |
| CA3018046A1 (en) | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| US20150166969A1 (en) | 2012-02-24 | 2015-06-18 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
| WO2013130824A1 (en) | 2012-02-29 | 2013-09-06 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| AU2013204327B2 (en) | 2012-04-20 | 2016-09-01 | Aviagen | Cell transfection method |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2013267209B2 (en) | 2012-06-01 | 2017-02-02 | Baruch S. Blumberg Institute | Modulation of hepatitis B virus cccDNA transcription |
| AU2013293270B2 (en) | 2012-07-25 | 2018-08-16 | Massachusetts Institute Of Technology | Inducible DNA binding proteins and genome perturbation tools and applications thereof |
| US8614194B1 (en) | 2012-07-25 | 2013-12-24 | Kaohsiung Medical University | Anionic cell penetrating peptide and its use for intracellular delivery |
| AU2013329186B2 (en) | 2012-10-10 | 2019-02-14 | Sangamo Therapeutics, Inc. | T cell modifying compounds and uses thereof |
| AU2013335451C1 (en) | 2012-10-23 | 2024-07-04 | Toolgen Incorporated | Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof |
| PL3360964T3 (pl) | 2012-12-06 | 2020-03-31 | Sigma-Aldrich Co. Llc | Modyfikacja i regulacja genomu oparta na crispr |
| WO2014093479A1 (en) | 2012-12-11 | 2014-06-19 | Montana State University | Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation |
| IL239344B2 (en) | 2012-12-12 | 2024-06-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| US20140186843A1 (en) | 2012-12-12 | 2014-07-03 | Massachusetts Institute Of Technology | Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CA2898184A1 (en) | 2013-01-16 | 2014-07-24 | Emory University | Cas9-nucleic acid complexes and uses related thereto |
| US11135273B2 (en) | 2013-02-07 | 2021-10-05 | The Rockefeller University | Sequence specific antimicrobials |
| US10660943B2 (en) | 2013-02-07 | 2020-05-26 | The Rockefeller University | Sequence specific antimicrobials |
| US9163837B2 (en) | 2013-02-27 | 2015-10-20 | Siemens Aktiengesellschaft | Flow conditioner in a combustor of a gas turbine engine |
| RU2662932C2 (ru) | 2013-03-14 | 2018-07-31 | Карибо Биосайенсиз, Инк. | Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты |
| US11332719B2 (en) | 2013-03-15 | 2022-05-17 | The Broad Institute, Inc. | Recombinant virus and preparations thereof |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| RU2619488C2 (ru) | 2013-03-27 | 2017-05-16 | Вилко Аг | Способ поточного обследования и/или тестирования устройств и аппарат для осуществления такого способа |
| CA2908253C (en) | 2013-04-04 | 2024-01-09 | Trustees Of Dartmouth College | Compositions and methods for in vivo excision of hiv-1 proviral dna |
| CN105683376A (zh) | 2013-05-15 | 2016-06-15 | 桑格摩生物科学股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| AU2014273082B2 (en) | 2013-05-29 | 2018-11-08 | Cellectis | A method for producing precise DNA cleavage using Cas9 nickase activity |
| SG10201913068PA (en) | 2013-06-04 | 2020-02-27 | Harvard College | Rna-guided transcriptional regulation |
| US9267135B2 (en) | 2013-06-04 | 2016-02-23 | President And Fellows Of Harvard College | RNA-guided transcriptional regulation |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| MX374532B (es) | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| RU2716420C2 (ru) | 2013-06-17 | 2020-03-11 | Те Брод Инститьют Инк. | Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени |
| CN105683379A (zh) | 2013-06-17 | 2016-06-15 | 布罗德研究所有限公司 | 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化 |
| EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| CN105492611A (zh) * | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| CN103343120B (zh) | 2013-07-04 | 2015-03-04 | 中国科学院遗传与发育生物学研究所 | 一种小麦基因组定点改造方法 |
| CA2917348A1 (en) | 2013-07-11 | 2015-01-15 | Moderna Therapeutics, Inc. | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
| CN103388006B (zh) | 2013-07-26 | 2015-10-28 | 华东师范大学 | 一种基因定点突变的构建方法 |
| JP7118588B2 (ja) | 2013-08-29 | 2022-08-16 | テンプル ユニヴァーシティ オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | Hiv感染のrnaガイド処置のための方法および組成物 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| WO2015048690A1 (en) | 2013-09-27 | 2015-04-02 | The Regents Of The University Of California | Optimized small guide rnas and methods of use |
| WO2015048577A2 (en) | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| WO2015065964A1 (en) | 2013-10-28 | 2015-05-07 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof |
| CN106459995B (zh) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| CA2930877A1 (en) | 2013-11-18 | 2015-05-21 | Crispr Therapeutics Ag | Crispr-cas system materials and methods |
| WO2015089427A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes |
| JP6712948B2 (ja) | 2013-12-12 | 2020-06-24 | ザ・ブロード・インスティテュート・インコーポレイテッド | 組成物、及びヌクレオチドリピート障害におけるcrispr−cas系の使用方法 |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| CN103668472B (zh) * | 2013-12-31 | 2014-12-24 | 北京大学 | 利用CRISPR/Cas9系统构建真核基因敲除文库的方法 |
| WO2015113063A1 (en) | 2014-01-27 | 2015-07-30 | Georgia Tech Research Corporation | Methods and systems for identifying crispr/cas off-target sites |
| JP6323228B2 (ja) | 2014-07-18 | 2018-05-16 | 富士電機株式会社 | 電力変換装置 |
| WO2016022866A1 (en) | 2014-08-07 | 2016-02-11 | Agilent Technologies, Inc. | Cis-blocked guide rna |
| WO2016028682A1 (en) | 2014-08-17 | 2016-02-25 | The Broad Institute Inc. | Genome editing using cas9 nickases |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| EP3711488A1 (en) | 2015-05-06 | 2020-09-23 | Snipr Technologies Limited | Altering microbial populations & modifying microbiota |
| 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 |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| JP6186470B2 (ja) | 2016-04-20 | 2017-08-23 | パイオニア株式会社 | 音響装置、音量制御方法、音量制御プログラム及び記録媒体 |
-
2014
- 2014-12-12 JP JP2016539070A patent/JP7103750B2/ja active Active
- 2014-12-12 KR KR1020167018585A patent/KR102805572B1/ko active Active
- 2014-12-12 CN CN202010044203.6A patent/CN111206032B/zh active Active
- 2014-12-12 CA CA2932478A patent/CA2932478A1/en active Pending
- 2014-12-12 SG SG10201804976YA patent/SG10201804976YA/en unknown
- 2014-12-12 EP EP14821438.0A patent/EP3079725B1/en active Active
- 2014-12-12 EP EP19200606.2A patent/EP3653229A1/en active Pending
- 2014-12-12 CN CN202410920804.7A patent/CN118813621A/zh active Pending
- 2014-12-12 IL IL316141A patent/IL316141A/en unknown
- 2014-12-12 CN CN201480072806.XA patent/CN106061510B/zh active Active
- 2014-12-12 KR KR1020257014984A patent/KR20250068794A/ko active Pending
- 2014-12-12 ES ES14821438T patent/ES2765481T3/es active Active
- 2014-12-12 DK DK14821438.0T patent/DK3079725T3/da active
- 2014-12-12 IL IL289736A patent/IL289736B2/en unknown
- 2014-12-12 MX MX2016007328A patent/MX374529B/es active IP Right Grant
- 2014-12-12 BR BR112016013201-7A patent/BR112016013201B1/pt active IP Right Grant
- 2014-12-12 AU AU2014361781A patent/AU2014361781B2/en active Active
- 2014-12-12 WO PCT/US2014/070127 patent/WO2015089462A1/en not_active Ceased
-
2016
- 2016-05-31 ZA ZA2016/03714A patent/ZA201603714B/en unknown
- 2016-06-06 MX MX2020008967A patent/MX2020008967A/es unknown
- 2016-06-08 IL IL246115A patent/IL246115B/en unknown
- 2016-06-10 US US15/179,799 patent/US11407985B2/en active Active
-
2020
- 2020-10-22 JP JP2020177177A patent/JP7689420B2/ja active Active
-
2021
- 2021-06-28 AU AU2021204395A patent/AU2021204395B2/en active Active
-
2022
- 2022-06-03 US US17/831,745 patent/US20230029506A1/en active Pending
-
2023
- 2023-06-16 JP JP2023098968A patent/JP2023116721A/ja active Pending
- 2023-08-22 AU AU2023219828A patent/AU2023219828B2/en active Active
-
2026
- 2026-03-12 AU AU2026201901A patent/AU2026201901A1/en not_active Withdrawn
- 2026-03-19 AU AU2026202160A patent/AU2026202160A1/en active Pending
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018051347A1 (en) * | 2016-09-14 | 2018-03-22 | Yeda Research And Development Co. Ltd. | Crisp-seq, an integrated method for massively parallel single cell rna-seq and crispr pooled screens |
| US11485971B2 (en) | 2016-09-14 | 2022-11-01 | Yeda Research And Development Co. Ltd. | CRISP-seq, an integrated method for massively parallel single cell RNA-seq and CRISPR pooled screens |
| CN109804069A (zh) * | 2016-09-23 | 2019-05-24 | ProQR治疗上市公司Ⅱ | 治疗眼部疾病的反义寡核苷酸 |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12571005B2 (en) | Protected guide RNAs (pgRNAs) | |
| JP7689420B2 (ja) | ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用 | |
| US20240093193A1 (en) | Dead guides for crispr transcription factors | |
| US12435320B2 (en) | CRISPR having or associated with destabilization domains | |
| US20170349894A1 (en) | Escorted and functionalized guides for crispr-cas systems | |
| CA2915845A1 (en) | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells | |
| CA2932475A1 (en) | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components | |
| CA2894681C (en) | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications | |
| CA2894681A1 (en) | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20191122 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION Effective date: 20240712 |
|
| B12 | Application deemed to be withdrawn, abandoned or lapsed |
Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION Effective date: 20240919 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 10TH ANNIV.) - STANDARD Year of fee payment: 10 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20241206 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20241218 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: N-6-6-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20250426 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: N-6-6-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20250426 |
|
| D22 | Grant of ip right intended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D22-D128 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALLOWANCE REQUIREMENTS DETERMINED COMPLIANT Effective date: 20251020 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251021 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 11TH ANNIV.) - STANDARD Year of fee payment: 11 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20251205 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20251205 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D00-D164 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: RESPONSE TO NOTICE OF ALLOWANCE Effective date: 20260105 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20260106 |
|
| D22 | Grant of ip right intended |
Free format text: ST27 STATUS EVENT CODE: A-2-4-D10-D22-D143 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRE-GRANT Effective date: 20260408 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20260408 |