IL244796A0 - Administration methods and compositions for nuclease-mediated genome engineering - Google Patents
Administration methods and compositions for nuclease-mediated genome engineeringInfo
- Publication number
- IL244796A0 IL244796A0 IL244796A IL24479616A IL244796A0 IL 244796 A0 IL244796 A0 IL 244796A0 IL 244796 A IL244796 A IL 244796A IL 24479616 A IL24479616 A IL 24479616A IL 244796 A0 IL244796 A0 IL 244796A0
- Authority
- IL
- Israel
- Prior art keywords
- nuclease
- compositions
- delivery methods
- genome engineering
- mediated genome
- Prior art date
Links
- 101710163270 Nuclease Proteins 0.000 title 1
- 238000002716 delivery method Methods 0.000 title 1
- 238000010362 genome editing Methods 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361892348P | 2013-10-17 | 2013-10-17 | |
| US201462033424P | 2014-08-05 | 2014-08-05 | |
| PCT/US2014/060931 WO2015057980A1 (en) | 2013-10-17 | 2014-10-16 | Delivery methods and compositions for nuclease-mediated genome engineering |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL244796A0 true IL244796A0 (en) | 2016-04-21 |
Family
ID=52826371
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL244796A IL244796A0 (en) | 2013-10-17 | 2016-03-28 | Administration methods and compositions for nuclease-mediated genome engineering |
| IL265426A IL265426B (en) | 2013-10-17 | 2019-03-18 | Administration methods and compositions for nuclease-mediated genome engineering |
| IL274182A IL274182B (en) | 2013-10-17 | 2020-04-23 | Administration methods and compositions for nuclease-mediated genome engineering |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL265426A IL265426B (en) | 2013-10-17 | 2019-03-18 | Administration methods and compositions for nuclease-mediated genome engineering |
| IL274182A IL274182B (en) | 2013-10-17 | 2020-04-23 | Administration methods and compositions for nuclease-mediated genome engineering |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US9957526B2 (enExample) |
| EP (2) | EP3441468B1 (enExample) |
| JP (5) | JP6646573B2 (enExample) |
| CN (3) | CN105899665B (enExample) |
| AU (3) | AU2014337248B2 (enExample) |
| CA (2) | CA3194037A1 (enExample) |
| DK (1) | DK3441468T3 (enExample) |
| ES (1) | ES2881473T3 (enExample) |
| IL (3) | IL244796A0 (enExample) |
| WO (1) | WO2015057980A1 (enExample) |
Families Citing this family (109)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| CA2908697C (en) | 2013-04-16 | 2023-12-12 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| 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 |
| JP2016536021A (ja) | 2013-11-07 | 2016-11-24 | エディタス・メディシン，インコーポレイテッド | ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物 |
| SG10201700961TA (en) | 2013-12-11 | 2017-04-27 | Regeneron Pharma | Methods and compositions for the targeted modification of a genome |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US9725710B2 (en) | 2014-01-08 | 2017-08-08 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| AU2015218576B2 (en) * | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| US9757420B2 (en) | 2014-07-25 | 2017-09-12 | Sangamo Therapeutics, Inc. | Gene editing for HIV gene therapy |
| CA2956224A1 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US12180263B2 (en) * | 2014-11-06 | 2024-12-31 | President And Fellows Of Harvard College | Cells lacking B2M surface expression and methods for allogeneic administration of such cells |
| EP3221457B1 (en) | 2014-11-21 | 2019-03-20 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| CN107921148A (zh) | 2015-05-08 | 2018-04-17 | 哈佛学院校长同事会 | 通用供体干细胞和相关方法 |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| CN108136047B (zh) * | 2015-05-13 | 2022-12-13 | 西雅图儿童医院（Dba西雅图儿童研究所） | 提高原代细胞中基于核酸内切酶的基因编辑 |
| WO2016201047A1 (en) | 2015-06-09 | 2016-12-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| WO2017053729A1 (en) * | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| DK3359184T3 (da) * | 2015-10-05 | 2020-06-15 | Prec Biosciences Inc | Genetisk modificerede celler, der omfatter et modificeret gen fra konstant alfaregion i human t-cellereceptor |
| DK3359660T3 (en) | 2015-10-05 | 2020-02-17 | Prec Biosciences Inc | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| EP4434589A3 (en) | 2015-10-23 | 2025-05-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| WO2017139264A1 (en) * | 2016-02-09 | 2017-08-17 | President And Fellows Of Harvard College | Dna-guided gene editing and regulation |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| CN109475109B (zh) | 2016-05-20 | 2021-10-29 | 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 |
| US10892034B2 (en) | 2016-07-01 | 2021-01-12 | Microsoft Technology Licensing, Llc | Use of homology direct repair to record timing of a molecular event |
| WO2018005117A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Storage through iterative dna editing |
| US11359234B2 (en) * | 2016-07-01 | 2022-06-14 | Microsoft Technology Licensing, Llc | Barcoding sequences for identification of gene expression |
| US20180004537A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
| US12385069B2 (en) * | 2016-07-22 | 2025-08-12 | The Board Of Regents Of The Nevada System Of Higher Education On Behalf Of The University Of Nevada | Compositions and methods for studying the Tat gene |
| RU2721125C1 (ru) | 2016-07-29 | 2020-05-18 | Регенерон Фармасьютикалз, Инк. | Мыши, содержащие мутации, вследствие которых экспрессируется укороченный на с-конце фибриллин-1 |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| SG11201901364VA (en) | 2016-08-24 | 2019-03-28 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| WO2018073393A2 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| GB2607227B (en) * | 2016-10-27 | 2023-05-10 | Intima Bioscience Inc | Viral methods of T cell therapy |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018136758A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hsd17b13 variants and uses thereof |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| WO2018176009A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| US20200392533A1 (en) * | 2017-04-11 | 2020-12-17 | President And Fellows Of Harvard College | In vivo gene editing of blood progenitors |
| AU2018254529B2 (en) | 2017-04-21 | 2024-03-28 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia |
| CA3059793A1 (en) | 2017-05-03 | 2018-11-08 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
| 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 |
| MX2019015188A (es) | 2017-06-15 | 2020-08-03 | Univ California | Inserciones de adn no virales orientadas. |
| US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
| CA3068465A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| EP3585160A2 (en) | 2017-07-31 | 2020-01-01 | Regeneron Pharmaceuticals, Inc. | Crispr reporter non-human animals and uses thereof |
| SG11201912235PA (en) | 2017-07-31 | 2020-01-30 | Regeneron Pharma | Cas-transgenic mouse embryonic stem cells and mice and uses thereof |
| AU2018309714A1 (en) | 2017-07-31 | 2020-01-30 | Regeneron Pharmaceuticals, Inc. | Assessment of CRISPR/Cas-induced recombination with an exogenous donor nucleic acid in vivo |
| WO2019032675A1 (en) | 2017-08-08 | 2019-02-14 | Sangamo Therapeutics, Inc. | CELL TARGETING MEDIATED BY A CHIMERIC ANTIGEN RECEPTOR |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| ES2962277T3 (es) | 2017-09-29 | 2024-03-18 | Regeneron Pharma | Roedores que comprenden un locus Ttr humanizado y métodos de uso |
| KR20200121782A (ko) | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| 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 |
| JP2021502085A (ja) | 2017-11-09 | 2021-01-28 | サンガモ セラピューティクス， インコーポレイテッド | サイトカイン誘導性ｓｈ２含有タンパク質（ｃｉｓｈ）遺伝子の遺伝子改変 |
| JP2021507561A (ja) | 2017-12-14 | 2021-02-22 | フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． | 音響トランスデューサドライバ及びコントローラ |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| US11786554B2 (en) | 2018-04-12 | 2023-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| JP2021533784A (ja) | 2018-08-18 | 2021-12-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | インサイチュ遺伝子編集 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| WO2020206139A1 (en) | 2019-04-04 | 2020-10-08 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized coagulation factor 12 locus |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3141159A1 (en) | 2019-05-21 | 2020-11-26 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| EP3801011A1 (en) | 2019-06-04 | 2021-04-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use |
| AU2020289581B2 (en) | 2019-06-07 | 2024-11-21 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized albumin locus |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| CN110628816B (zh) * | 2019-09-12 | 2022-10-18 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 一种用于血友病a的双切供体及其药物组合 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021108363A1 (en) | 2019-11-25 | 2021-06-03 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-mediated upregulation of humanized ttr allele |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| US12250931B2 (en) | 2020-01-28 | 2025-03-18 | Regeneron Pharmaceuticals, Inc. | Genetically modified mouse with a humanized PNPLA3 gene and methods of use |
| EP4099821A1 (en) | 2020-02-07 | 2022-12-14 | Regeneron Pharmaceuticals, Inc. | <smallcaps/>? ? ?klkb1? ? ? ? ?non-human animals comprising a humanizedlocus and methods of use |
| US20230112792A1 (en) * | 2020-03-13 | 2023-04-13 | Pairwise Plants Services, Inc. | Methods of introducing variation into plants and products produced therefrom |
| WO2021195079A1 (en) | 2020-03-23 | 2021-09-30 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use |
| CA3177093A1 (en) | 2020-05-06 | 2021-11-11 | Cellectis S.A. | Methods for targeted insertion of exogenous sequences in cellular genomes |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| US20240018493A1 (en) * | 2020-11-10 | 2024-01-18 | The Board Of Trustees Of The Leland Stanford Junior University | Knock-in of large dna for long-term high genomic expression |
| KR20240099259A (ko) | 2021-10-14 | 2024-06-28 | 아스널 바이오사이언시스, 인크. | 공동 발현된 shrna 및 논리 게이트 시스템을 갖는 면역 세포 |
| EP4423271A2 (en) | 2021-10-28 | 2024-09-04 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-related methods and compositions for knocking out c5 |
| KR20240117571A (ko) | 2021-12-08 | 2024-08-01 | 리제너론 파마슈티칼스 인코포레이티드 | 돌연변이 마이오실린 질환 모델 및 이의 용도 |
| JP2025508677A (ja) | 2022-02-02 | 2025-04-10 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ポンペ病の処置のための抗ＴｆＲ：ＧＡＡ及び抗ＣＤ６３：ＧＡＡの挿入 |
| EP4514981A2 (en) | 2022-04-29 | 2025-03-05 | Regeneron Pharmaceuticals, Inc. | Identification of tissue-specific extragenic safe harbors for gene therapy approaches |
| WO2023235725A2 (en) | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr-based therapeutics for c9orf72 repeat expansion disease |
| EP4532720A2 (en) | 2022-05-31 | 2025-04-09 | Regeneron Pharmaceuticals, Inc. | Crispr interference therapeutics for c9orf72 repeat expansion disease |
| AU2024315073A1 (en) | 2023-07-28 | 2026-01-22 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
| WO2025170018A1 (ja) * | 2024-02-09 | 2025-08-14 | 国立大学法人広島大学 | ジンクフィンガータンパク質及びその組み合わせ、ジンクフィンガーヌクレアーゼ及びジンクフィンガーヌクレアーゼ対、標的ｄｎａの編集方法、標的ｄｎａが編集された細胞の製造方法、及びキット |
| CN120099099A (zh) * | 2024-03-05 | 2025-06-06 | 百奥赛图(北京)医药科技股份有限公司 | 一种trbc基因修饰的非人动物 |
| US20250388890A1 (en) | 2024-06-20 | 2025-12-25 | Regeneron Pharmaceuticals, Inc. | ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I |
Family Cites Families (102)
| 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 |
| 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 |
| 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 |
| 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 |
| US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| AU7979491A (en) | 1990-05-03 | 1991-11-27 | 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 |
| US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| 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 |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| CA2181548C (en) | 1994-01-18 | 2009-11-03 | Carlos F. Barbas, Iii | Zinc finger protein derivatives and methods therefor |
| JP4285766B2 (ja) | 1994-03-23 | 2009-06-24 | オハイオ ユニバーシティ | 緻密にした核酸および細胞へのデリバリ |
| US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| DE69535829D1 (de) | 1994-08-20 | 2008-10-16 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von dna |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| US6342345B1 (en) | 1997-04-02 | 2002-01-29 | The Board Of Trustees Of The Leland Stanford Junior University | Detection of molecular interactions by reporter subunit complementation |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
| US6140815A (en) | 1998-06-17 | 2000-10-31 | Dover Instrument Corporation | High stability spin stand platform |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| CA2394850C (en) | 1999-12-06 | 2012-02-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| EP1254369B1 (en) | 2000-02-08 | 2010-10-06 | Sangamo BioSciences, Inc. | Cells for drug discovery |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| AU2001263155A1 (en) | 2000-05-16 | 2001-11-26 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| WO2003016496A2 (en) | 2001-08-20 | 2003-02-27 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| GB0210032D0 (en) | 2002-05-02 | 2002-06-12 | Ibm | Method for ordering parallel operations in a resource manager |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| WO2004037977A2 (en) | 2002-09-05 | 2004-05-06 | California Institute Of Thechnology | Use of chimeric nucleases to stimulate gene targeting |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| JP4903689B2 (ja) | 2004-04-08 | 2012-03-28 | サンガモ バイオサイエンシズ インコーポレイテッド | 神経障害および神経変性症状を治療するための方法と組成物 |
| US7253273B2 (en) | 2004-04-08 | 2007-08-07 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| AU2005287278B2 (en) | 2004-09-16 | 2011-08-04 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| WO2006121866A2 (en) | 2005-05-05 | 2006-11-16 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| EP1913149A4 (en) | 2005-07-26 | 2009-08-05 | Sangamo Biosciences Inc | TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES |
| WO2007030674A2 (en) * | 2005-09-07 | 2007-03-15 | Maxcyte, Inc. | Use of nucleases to improve viability and enhance transgene expression in transfected cells |
| CA2626262C (en) | 2005-10-18 | 2015-09-08 | Homme W. Hellinga | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| EP2019839B1 (en) * | 2006-05-25 | 2011-12-07 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| DE602007005634D1 (de) | 2006-05-25 | 2010-05-12 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
| WO2008109467A1 (en) | 2007-03-02 | 2008-09-12 | Arizona Board Of Regents For And On Behalf Of Arizona State University | Electrically conducting porphyrin and porphyrin-fullerene electropolymers |
| US8110379B2 (en) * | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
| DK2205749T3 (en) | 2007-09-27 | 2016-08-22 | Dow Agrosciences Llc | MODIFIED PROTEINS zinc finger, which target the 5-enolpyruvylshikimate-3-phosphate synthase genes |
| US8563314B2 (en) | 2007-09-27 | 2013-10-22 | Sangamo Biosciences, Inc. | Methods and compositions for modulating PD1 |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| JP5681114B2 (ja) | 2008-12-04 | 2015-03-04 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集 |
| ES2654923T3 (es) | 2008-12-17 | 2018-02-15 | Dow Agrosciences Llc | Integración dirigida en la localización Zp15 |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| CA2951341A1 (en) * | 2009-12-21 | 2011-06-30 | Keygene N.V. | Improved techniques for transfecting protoplasts |
| DK2534173T3 (da) | 2010-02-08 | 2019-10-14 | Sangamo Therapeutics Inc | Manipulerede spaltnings-halvdomæner |
| EP2534163B1 (en) | 2010-02-09 | 2015-11-04 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US9315825B2 (en) * | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| KR101880536B1 (ko) | 2010-04-26 | 2018-07-23 | 상가모 테라퓨틱스, 인코포레이티드 | 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집 |
| KR101974036B1 (ko) | 2010-05-03 | 2019-04-30 | 상가모 테라퓨틱스, 인코포레이티드 | 아연 핑거 모듈을 연결하기 위한 조성물 |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
| WO2012021632A2 (en) * | 2010-08-10 | 2012-02-16 | The Johns Hopkins University | Generation and use of pluripotent stem cells |
| CN103270046B (zh) * | 2010-10-12 | 2016-05-11 | 费城儿童医院 | 治疗b型血友病的方法和组合物 |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| CA2848417C (en) * | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA2852955C (en) * | 2011-10-27 | 2021-02-16 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| CA2871524C (en) | 2012-05-07 | 2021-07-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| EP2872154B1 (en) * | 2012-07-11 | 2017-05-31 | Sangamo BioSciences, Inc. | Methods and compositions for delivery of biologics |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| EP4282970A3 (en) * | 2012-12-17 | 2024-01-17 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| CN116083487A (zh) | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| AU2015218576B2 (en) | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
-
2014
- 2014-10-16 CN CN201480055149.8A patent/CN105899665B/zh not_active Expired - Fee Related
- 2014-10-16 JP JP2016523272A patent/JP6646573B2/ja not_active Expired - Fee Related
- 2014-10-16 EP EP18192631.2A patent/EP3441468B1/en active Active
- 2014-10-16 CA CA3194037A patent/CA3194037A1/en active Pending
- 2014-10-16 DK DK18192631.2T patent/DK3441468T3/da active
- 2014-10-16 CN CN202310865224.8A patent/CN116836957A/zh active Pending
- 2014-10-16 CA CA2926094A patent/CA2926094C/en active Active
- 2014-10-16 AU AU2014337248A patent/AU2014337248B2/en not_active Ceased
- 2014-10-16 WO PCT/US2014/060931 patent/WO2015057980A1/en not_active Ceased
- 2014-10-16 EP EP14854505.6A patent/EP3058072B1/en active Active
- 2014-10-16 CN CN201910993837.3A patent/CN110713995B/zh not_active Expired - Fee Related
- 2014-10-16 ES ES18192631T patent/ES2881473T3/es active Active
- 2014-10-16 US US14/516,236 patent/US9957526B2/en active Active
-
2016
- 2016-03-28 IL IL244796A patent/IL244796A0/en unknown
-
2018
- 2018-03-26 US US15/935,908 patent/US10494648B2/en active Active
- 2018-12-25 JP JP2018240821A patent/JP6875362B2/ja not_active Expired - Fee Related
- 2018-12-25 JP JP2018240822A patent/JP2019041777A/ja active Pending
-
2019
- 2019-03-18 IL IL265426A patent/IL265426B/en active IP Right Grant
-
2020
- 2020-04-23 IL IL274182A patent/IL274182B/en unknown
- 2020-08-07 AU AU2020213379A patent/AU2020213379B2/en not_active Ceased
- 2020-11-02 JP JP2020183610A patent/JP2021010383A/ja not_active Withdrawn
-
2022
- 2022-11-02 JP JP2022176277A patent/JP2022190071A/ja active Pending
- 2022-11-17 AU AU2022271447A patent/AU2022271447A1/en not_active Abandoned
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL274182B (en) | Administration methods and compositions for nuclease-mediated genome engineering | |
| IL277196A (en) | Methods and preparations for genome engineering | |
| IL272193B (en) | Genome engineering | |
| IL269461B (en) | Cytidine-5-carboxamide modified nucleotide preparations and related methods | |
| IL273205A (en) | Preparations and methods | |
| ZA201506682B (en) | Compositions and methods for the improved production and delivery of rna | |
| IL245674A0 (en) | Methods and preparations for the targeted modification of a genome | |
| GB2521022B (en) | Compositions and methods | |
| IL244081A0 (en) | Preparations and methods for modulating RNA | |
| SMT201900635T1 (it) | Composizioni e metodi di attivazione della segnalazione dipendente dallo "stimolatore di geni interferone" | |
| GB201308072D0 (en) | Compositions and methods | |
| SG11201602331QA (en) | Compositions and methods utilizing lysophosphatidylcholine scaffolds | |
| IL244082A0 (en) | Compositions and methods for modulating DNA methylation | |
| GB201305813D0 (en) | Compositions and methods | |
| SG11201509017VA (en) | Compositions and methods for nutrient delivery | |
| HRP20181632T1 (hr) | Postupci i kompoziti za ciljanu modificiranje genoma | |
| GB201322617D0 (en) | Methods and compositions | |
| GB201312393D0 (en) | Compositions and Methods |