HK1198178A1 - Methods and compositions for regulation of transgene expression - Google Patents
Methods and compositions for regulation of transgene expression Download PDFInfo
- Publication number
- HK1198178A1 HK1198178A1 HK14111232.9A HK14111232A HK1198178A1 HK 1198178 A1 HK1198178 A1 HK 1198178A1 HK 14111232 A HK14111232 A HK 14111232A HK 1198178 A1 HK1198178 A1 HK 1198178A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- cell
- albumin
- gene
- protein
- sequence
- Prior art date
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
- 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
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
-
- 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
-
- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/067—Hepatocytes
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
-
- 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
- C12N2510/00—Genetically modified 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Transplantation (AREA)
- Pulmonology (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Cell Biology (AREA)
- Epidemiology (AREA)
- Endocrinology (AREA)
- Emergency Medicine (AREA)
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201161537349P | 2011-09-21 | 2011-09-21 | |
US61/537,349 | 2011-09-21 | ||
US201161560506P | 2011-11-16 | 2011-11-16 | |
US61/560,506 | 2011-11-16 | ||
US201261670490P | 2012-07-11 | 2012-07-11 | |
US61/670,490 | 2012-07-11 | ||
PCT/US2012/056539 WO2013044008A2 (en) | 2011-09-21 | 2012-09-21 | Methods and compositions for regulation of transgene expression |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1198178A1 true HK1198178A1 (en) | 2015-03-13 |
Family
ID=47915095
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK14111232.9A HK1198178A1 (en) | 2011-09-21 | 2012-09-21 | Methods and compositions for regulation of transgene expression |
Country Status (12)
Country | Link |
---|---|
US (6) | US9394545B2 (xx) |
EP (2) | EP3498833B1 (xx) |
JP (3) | JP6185916B2 (xx) |
KR (2) | KR102061557B1 (xx) |
CN (2) | CN108610423A (xx) |
AU (3) | AU2012312260B2 (xx) |
CA (2) | CA2848417C (xx) |
ES (1) | ES2961613T3 (xx) |
HK (1) | HK1198178A1 (xx) |
IL (5) | IL277027B (xx) |
SG (2) | SG10201811736QA (xx) |
WO (1) | WO2013044008A2 (xx) |
Families Citing this family (162)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9394545B2 (en) | 2011-09-21 | 2016-07-19 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
LT2800811T (lt) | 2012-05-25 | 2017-09-11 | The Regents Of The University Of California | Būdai ir kompozicijos, skirti tikslinės dnr modifikavimui, panaudojant adresuotą rnr, ir transkripcijos moduliavimui, panaudojant adresuotą rnr |
EP2872154B1 (en) | 2012-07-11 | 2017-05-31 | Sangamo BioSciences, Inc. | Methods and compositions for delivery of biologics |
US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
WO2014011237A1 (en) | 2012-07-11 | 2014-01-16 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
UA118957C2 (uk) | 2012-08-29 | 2019-04-10 | Сангамо Біосайєнсиз, Інк. | Спосіб і композиція для лікування генетичного захворювання |
WO2014059173A2 (en) | 2012-10-10 | 2014-04-17 | Sangamo Biosciences, Inc. | T cell modifying compounds and uses thereof |
MX363017B (es) * | 2012-11-01 | 2019-03-04 | Factor Bioscience Inc | Métodos y productos para la expresión de proteínas en células. |
CA2892448A1 (en) * | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
EP3138911B1 (en) | 2012-12-06 | 2018-12-05 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
EP2931892B1 (en) | 2012-12-12 | 2018-09-12 | 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 |
US20140179770A1 (en) | 2012-12-12 | 2014-06-26 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
WO2014130955A1 (en) | 2013-02-25 | 2014-08-28 | Sangamo Biosciences, Inc. | Methods and compositions for enhancing nuclease-mediated gene disruption |
WO2014134412A1 (en) | 2013-03-01 | 2014-09-04 | Regents Of The University Of Minnesota | Talen-based gene correction |
ES2749624T3 (es) | 2013-03-14 | 2020-03-23 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
JP6346266B2 (ja) | 2013-03-21 | 2018-06-20 | サンガモ セラピューティクス, インコーポレイテッド | 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するt細胞受容体遺伝子の標的化された破壊 |
JP2016518142A (ja) * | 2013-05-10 | 2016-06-23 | サンガモ バイオサイエンシーズ, インコーポレイテッド | ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物 |
EP3730615A3 (en) | 2013-05-15 | 2020-12-09 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
KR20160030187A (ko) * | 2013-06-17 | 2016-03-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 간의 표적화 및 치료를 위한 CRISPRCas 시스템, 벡터 및 조성물의 전달 및 용도 |
EP3011030B1 (en) | 2013-06-17 | 2023-11-08 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
KR20160044457A (ko) | 2013-06-17 | 2016-04-25 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작을 위한 탠덤 안내 시스템, 방법 및 조성물의 전달, 조작 및 최적화 |
WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
BR122021009076B1 (pt) | 2013-06-17 | 2024-02-15 | The Broad Institute Inc. | Vetor viral contendo molécula(s) de ácido nucleico heterólogo, composição, uso e métodos do mesmo |
DK3011032T3 (da) * | 2013-06-17 | 2020-01-20 | Broad Inst Inc | Fremføring, modificering og optimering af systemer, fremgangsmåder og sammensætninger til målretning mod og modellering af sygdomme og forstyrrelser i postmitotiske celler |
CN105555132B (zh) * | 2013-07-10 | 2018-07-17 | 约瑟夫·A·玛吉朱布 | Mrap2敲除 |
JP6588438B2 (ja) | 2013-08-28 | 2019-10-09 | サンガモ セラピューティクス, インコーポレイテッド | Dna結合ドメインと切断ドメインとを連結するための組成物 |
WO2015057976A1 (en) | 2013-10-17 | 2015-04-23 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
EP3058072B1 (en) * | 2013-10-17 | 2021-05-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
US10369201B2 (en) | 2013-11-11 | 2019-08-06 | Sangamo Therapeutics, Inc. | Methods and compositions for treating Huntington's disease |
PT3492593T (pt) | 2013-11-13 | 2021-10-18 | Childrens Medical Center | Regulação da expressão de genes mediada por nucleases |
AU2014364051B2 (en) | 2013-12-09 | 2018-12-20 | Sangamo Therapeutics, Inc. | Methods and compositions for treating hemophilia |
WO2015089354A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
ES2765481T3 (es) | 2013-12-12 | 2020-06-09 | Broad Inst Inc | Administración, uso y aplicaciones terapéuticas de los sistemas crispr-cas y composiciones para la edición genómica |
RU2016128077A (ru) | 2013-12-12 | 2018-12-06 | Те Брод Инститьют Инк. | Доставка, применение и применения в терапии систем и композиций crispr-cas для лечения обусловленных hbv и вирусных заболеваний и нарушений |
LT3102673T (lt) | 2014-02-03 | 2020-08-25 | Sangamo Therapeutics, Inc. | Beta talasemijos gydymo būdai ir kompozicijos |
WO2015127439A1 (en) | 2014-02-24 | 2015-08-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration |
CA2942762C (en) | 2014-03-18 | 2023-10-17 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
WO2015164748A1 (en) | 2014-04-24 | 2015-10-29 | Sangamo Biosciences, Inc. | Engineered transcription activator like effector (tale) proteins |
AU2015255877B2 (en) | 2014-05-08 | 2020-03-26 | Chdi Foundation, Inc. | Methods and compositions for treating huntington's disease |
WO2015175642A2 (en) * | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
EP3152319A4 (en) | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
WO2016014794A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
WO2016014837A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Gene editing for hiv gene therapy |
US9616090B2 (en) | 2014-07-30 | 2017-04-11 | Sangamo Biosciences, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
SI3194570T1 (sl) | 2014-09-16 | 2022-01-31 | Sangamo Therapeutics, Inc. | Postopki in sestavki za genomski inženiring s posredovanjem nukleaze in korekcijo krvotvornih matičnih celic |
EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
US10889834B2 (en) | 2014-12-15 | 2021-01-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing targeted transgene integration |
WO2016118726A2 (en) | 2015-01-21 | 2016-07-28 | Sangamo Biosciences, Inc. | Methods and compositions for identification of highly specific nucleases |
US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
JP6873917B2 (ja) | 2015-05-12 | 2021-05-19 | サンガモ セラピューティクス, インコーポレイテッド | ヌクレアーゼ介在性遺伝子発現調節 |
WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
WO2016205613A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Crispr enzyme mutations reducing off-target effects |
US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
CN108024544B (zh) | 2015-07-13 | 2022-04-29 | 桑格摩生物治疗股份有限公司 | 用于核酸酶介导的基因组工程化的递送方法及组合物 |
WO2017053753A1 (en) | 2015-09-23 | 2017-03-30 | Sangamo Biosciences, Inc. | Htt repressors and uses thereof |
AU2016343887B2 (en) * | 2015-10-28 | 2023-04-06 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
LT3368507T (lt) | 2015-10-28 | 2023-03-10 | Acuitas Therapeutics Inc. | Nauji lipidai ir lipidų nanodalelių kompozicijos, skirtos nukleorūgščių tiekimui |
US20200208173A1 (en) * | 2015-10-30 | 2020-07-02 | Seracare Life Sciences, Inc. | Adenovirus control virus |
AU2016361350B2 (en) * | 2015-11-23 | 2023-04-06 | Sangamo Therapeutics, Inc. | Methods and compositions for engineering immunity |
US11389546B2 (en) | 2015-12-09 | 2022-07-19 | Modernatx, Inc. | Heterologous UTR sequences for enhanced mRNA expression |
EA201891212A1 (ru) | 2015-12-18 | 2019-01-31 | Сангамо Терапьютикс, Инк. | Адресная дезорганизация клеточного рецептора гкгс |
WO2017106528A2 (en) | 2015-12-18 | 2017-06-22 | Sangamo Biosciences, Inc. | Targeted disruption of the t cell receptor |
KR20180127319A (ko) | 2016-01-15 | 2018-11-28 | 상가모 테라퓨틱스, 인코포레이티드 | 신경 질환의 치료를 위한 방법 및 조성물 |
CN109069568B (zh) | 2016-02-02 | 2023-07-07 | 桑格摩生物治疗股份有限公司 | 用于连接dna结合结构域和切割结构域的组合物 |
CA3033788A1 (en) * | 2016-08-17 | 2018-02-22 | Factor Bioscience Inc. | Nucleic acid products and methods of administration thereof |
BR112019003100A2 (pt) | 2016-08-24 | 2019-07-09 | Sangamo Therapeutics Inc | regulação da expressão gênica usando nucleases manipuladas |
CA3034884A1 (en) | 2016-08-24 | 2018-03-01 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
JP7256739B2 (ja) | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス, インコーポレイテッド | 肝臓遺伝子のモジュレーション |
JP7042263B2 (ja) | 2016-10-20 | 2022-03-25 | サンガモ セラピューティクス, インコーポレイテッド | ファブリー病の治療のための方法および組成物 |
WO2018081775A1 (en) | 2016-10-31 | 2018-05-03 | Sangamo Therapeutics, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
JP2020500539A (ja) * | 2016-12-09 | 2020-01-16 | サンガモ セラピューティクス, インコーポレイテッド | 標的特異的ヌクレアーゼの送達 |
WO2018126095A1 (en) * | 2016-12-29 | 2018-07-05 | Applied Stemcell, Inc. | Transgenic animals and method for bioproduction |
WO2018191657A1 (en) | 2017-04-13 | 2018-10-18 | Acuitas Therapeutics, Inc. | Lipids for delivery of active agents |
CA3061612A1 (en) | 2017-04-28 | 2018-11-01 | Acuitas Therapeutics, Inc. | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
WO2018204469A2 (en) | 2017-05-03 | 2018-11-08 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
CA3067316A1 (en) | 2017-06-15 | 2018-12-20 | Toolgen Incorporated | Platform for expressing protein of interest in liver |
US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
KR102712142B1 (ko) | 2017-07-31 | 2024-10-07 | 리제너론 파마슈티칼스 인코포레이티드 | Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도 |
JP2020533957A (ja) | 2017-07-31 | 2020-11-26 | 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Regeneron Pharmaceuticals, Inc. | Crisprリポーター非ヒト動物およびその使用 |
KR20200032117A (ko) | 2017-07-31 | 2020-03-25 | 리제너론 파마슈티칼스 인코포레이티드 | 생체 내에서 외인성 공여체 핵산과의 CRISPR/Cas-유도된 재조합의 평가 |
WO2019036008A1 (en) | 2017-08-16 | 2019-02-21 | Acuitas Therapeutics, Inc. | LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS |
US11542225B2 (en) | 2017-08-17 | 2023-01-03 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
US11524932B2 (en) | 2017-08-17 | 2022-12-13 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
JP7461872B2 (ja) | 2017-08-17 | 2024-04-04 | アクイタス セラピューティクス インコーポレイテッド | 脂質ナノ粒子製剤における使用のための脂質 |
AU2018353012B2 (en) * | 2017-10-17 | 2025-03-27 | Bayer Healthcare Llc | Compositions and methods for gene editing for Hemophilia A |
WO2019084140A1 (en) * | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES |
WO2019094725A2 (en) | 2017-11-09 | 2019-05-16 | Sangamo Therapeutics, Inc. | Genetic modification of cytokine inducible sh2-containing protein (cish) gene |
WO2019157324A1 (en) | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
WO2019161133A1 (en) | 2018-02-15 | 2019-08-22 | Memorial Sloan Kettering Cancer Center | Foxp3 targeting agent compositions and methods of use for adoptive cell therapy |
CN116349651A (zh) | 2018-03-19 | 2023-06-30 | 瑞泽恩制药公司 | 使用crispr/cas系统对动物进行转录调制 |
MA52656A (fr) | 2018-04-05 | 2021-02-17 | Editas Medicine Inc | Procédés de production de cellules exprimant un récepteur recombinant et compositions associées |
CN112585277A (zh) | 2018-04-05 | 2021-03-30 | 朱诺治疗学股份有限公司 | 表达重组受体的t细胞、相关多核苷酸和方法 |
CN112313246B (zh) | 2018-04-18 | 2025-05-13 | 桑格摩生物治疗股份有限公司 | 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物 |
EP3781193A4 (en) | 2018-04-18 | 2022-01-26 | Altius Institute For Biomedical Sciences | Animal pathogen-derived polypeptides and uses thereof for genetic engineering |
US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
WO2020006126A1 (en) | 2018-06-27 | 2020-01-02 | Altius Institute For Biomedical Sciences | Nucleic acid binding domains and methods of use thereof |
CN112533645A (zh) * | 2018-08-03 | 2021-03-19 | 桑格摩生物治疗股份有限公司 | 通过因子viii的表达改善临床参数 |
EP3841204A4 (en) | 2018-08-23 | 2022-05-18 | Sangamo Therapeutics, Inc. | CHANGED TARGET SPECIFIC BASE EDITORS |
WO2020056170A1 (en) | 2018-09-12 | 2020-03-19 | Fred Hutchinson Cancer Research Center | Reducing cd33 expression to selectively protect therapeutic cells |
WO2020061161A1 (en) | 2018-09-18 | 2020-03-26 | Sangamo Therapeutics, Inc. | Programmed cell death 1 (pd1) specific nucleases |
WO2020061426A2 (en) | 2018-09-21 | 2020-03-26 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
WO2020069029A1 (en) | 2018-09-26 | 2020-04-02 | Emendobio Inc. | Novel crispr nucleases |
TW202028461A (zh) | 2018-10-18 | 2020-08-01 | 美商英特利亞醫療公司 | 核酸構築體及使用方法 |
TW202507011A (zh) * | 2018-10-18 | 2025-02-16 | 美商英特利亞醫療公司 | 用於從白蛋白基因座表現轉殖基因的組成物及方法 |
SG11202103734PA (en) | 2018-10-18 | 2021-05-28 | Intellia Therapeutics Inc | Compositions and methods for expressing factor ix |
CN113166727A (zh) | 2018-11-28 | 2021-07-23 | 四十七公司 | 对消融方案耐受的基因修饰的hspc |
PT3908568T (pt) | 2019-01-11 | 2024-09-30 | Acuitas Therapeutics Inc | Lípidos para a administração de agentes ativos por nanopartículas lipídicas |
US20220098621A1 (en) | 2019-02-05 | 2022-03-31 | Emendobio Inc. | Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene |
US20220154157A1 (en) | 2019-02-06 | 2022-05-19 | Emendobio Inc. | New engineered high fidelity cas9 |
KR20210148154A (ko) | 2019-04-03 | 2021-12-07 | 리제너론 파마슈티칼스 인코포레이티드 | 세이프 하버 좌위 내로의 항체 코딩 서열의 삽입을 위한 방법 및 조성물 |
AU2020265741A1 (en) | 2019-05-01 | 2021-11-25 | Editas Medicine, Inc. | Cells expressing a recombinant receptor from a modified TGFBR2 Locus, related polynucleotides and methods |
AU2020265749A1 (en) | 2019-05-01 | 2022-01-06 | Juno Therapeutics, Inc. | Cells expressing a chimeric receptor from a modified CD247 locus, related polynucleotides and methods |
BR112021022722A2 (pt) | 2019-06-07 | 2022-01-04 | Regeneron Pharma | Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente |
EP3994270A1 (en) | 2019-07-02 | 2022-05-11 | Fred Hutchinson Cancer Research Center | Recombinant ad35 vectors and related gene therapy improvements |
WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
WO2021092513A1 (en) | 2019-11-08 | 2021-05-14 | Regeneron Pharmaceuticals, Inc. | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
WO2021108363A1 (en) | 2019-11-25 | 2021-06-03 | Regeneron Pharmaceuticals, Inc. | Crispr/cas-mediated upregulation of humanized ttr allele |
CN111088282B (zh) * | 2020-03-23 | 2020-08-28 | 上海安民生物技术有限公司 | Aavs1和h11安全港位点在重组表达蛋白中的应用 |
EP4125348A1 (en) | 2020-03-23 | 2023-02-08 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use |
CN113061169B (zh) * | 2020-03-24 | 2022-09-27 | 江南大学 | 一种转录调控蛋白及其在共轭亚油酸生产中的应用 |
EP4150057A2 (en) | 2020-05-13 | 2023-03-22 | Juno Therapeutics, Inc. | Process for producing donor-batched cells expressing a recombinant receptor |
JP2023531531A (ja) | 2020-06-26 | 2023-07-24 | ジュノ セラピューティクス ゲーエムベーハー | 組換え受容体を条件付きで発現する操作されたt細胞、関連ポリヌクレオチド、および方法 |
KR20230051172A (ko) | 2020-07-16 | 2023-04-17 | 아퀴타스 테라퓨틱스 인크. | 지질 나노 입자에 사용하기 위한 양이온성 지질 |
EP4240756A1 (en) | 2020-11-04 | 2023-09-13 | Juno Therapeutics, Inc. | Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods |
CA3213080A1 (en) | 2021-03-23 | 2022-09-29 | Krit RITTHIPICHAI | Cish gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy |
KR20220136677A (ko) * | 2021-04-01 | 2022-10-11 | 서울대학교산학협력단 | 인간 면역 글로불린 Fc 및 목적단백질을 생산하는 조류의 제조방법 |
TW202302846A (zh) * | 2021-04-16 | 2023-01-16 | 中國大陸商杭州啟函生物科技有限公司 | 用於細胞工程化的安全港基因座 |
WO2023035011A1 (en) | 2021-09-03 | 2023-03-09 | North Carolina State University | Compositions and methods for conferring resistance to geminivirus |
WO2023077012A1 (en) | 2021-10-27 | 2023-05-04 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
EP4426832A1 (en) | 2021-11-03 | 2024-09-11 | The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone | Precise genome editing using retrons |
WO2023081900A1 (en) | 2021-11-08 | 2023-05-11 | Juno Therapeutics, Inc. | Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods |
CN118632622A (zh) | 2021-12-08 | 2024-09-10 | 瑞泽恩制药公司 | 突变型肌纤蛋白疾病模型及其用途 |
CA3242402A1 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
MX2024008275A (es) | 2021-12-30 | 2024-09-18 | Regel Therapeutics Inc | Composiciones para modular la expresion de la subunidad alfa 1 del canal activado por voltaje de sodio y sus usos. |
WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
KR20240135629A (ko) | 2022-02-02 | 2024-09-11 | 리제너론 파마슈티칼스 인코포레이티드 | 폼페병의 치료를 위한 항-TfR:GAA 및 항-CD63:GAA 삽입 |
EP4476331A2 (en) | 2022-02-07 | 2024-12-18 | Ensoma, Inc. | Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids |
WO2023150798A1 (en) | 2022-02-07 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for defining optimal treatment timeframes in lysosomal disease |
EP4476245A1 (en) | 2022-02-11 | 2024-12-18 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for screening 4r tau targeting agents |
EP4522203A1 (en) | 2022-05-09 | 2025-03-19 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified capsid for gene therapy |
WO2023220043A1 (en) | 2022-05-09 | 2023-11-16 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified genome for gene therapy |
EP4522201A1 (en) | 2022-05-09 | 2025-03-19 | Synteny Therapeutics, Inc. | Erythroparvovirus compositions and methods for gene therapy |
EP4532707A2 (en) * | 2022-05-25 | 2025-04-09 | Metagenomi, Inc. | Supplementation of liver enzyme expression |
AU2023314808A1 (en) | 2022-07-29 | 2025-03-20 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle |
WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
KR20250075694A (ko) | 2022-09-28 | 2025-05-28 | 리제너론 파마슈티칼스 인코포레이티드 | 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체 |
WO2024098002A1 (en) | 2022-11-04 | 2024-05-10 | Regeneron Pharmaceuticals, Inc. | Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle |
WO2024100604A1 (en) | 2022-11-09 | 2024-05-16 | Juno Therapeutics Gmbh | Methods for manufacturing engineered immune cells |
WO2024107765A2 (en) | 2022-11-14 | 2024-05-23 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes |
WO2024161021A1 (en) | 2023-02-03 | 2024-08-08 | Juno Therapeutics Gmbh | Methods for non-viral manufacturing of engineered immune cells |
WO2024196965A1 (en) | 2023-03-23 | 2024-09-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
WO2024197242A1 (en) | 2023-03-23 | 2024-09-26 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods |
WO2024249172A1 (en) | 2023-05-26 | 2024-12-05 | Regel Therapeutics, Inc. | Compositions for modulating expression of sodium voltage-gated channel alpha subunit 2 and uses thereof |
TW202513801A (zh) | 2023-07-28 | 2025-04-01 | 美商雷傑納榮製藥公司 | 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入 |
US20250049896A1 (en) | 2023-07-28 | 2025-02-13 | Regeneron Pharmaceuticals, Inc. | Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency |
WO2025029654A2 (en) | 2023-07-28 | 2025-02-06 | Regeneron Pharmaceuticals, Inc. | Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion |
WO2025049524A1 (en) | 2023-08-28 | 2025-03-06 | Regeneron Pharmaceuticals, Inc. | Cxcr4 antibody-resistant modified receptors |
WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
WO2025132815A1 (en) | 2023-12-20 | 2025-06-26 | Novozymes A/S | Novel cas nucleases and polynucleotides encoding the same |
Family Cites Families (104)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US789538A (en) | 1904-11-11 | 1905-05-09 | Colin E Ham | Dumb-bell. |
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 |
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 |
US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
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 |
AU704601B2 (en) | 1994-01-18 | 1999-04-29 | Scripps Research Institute, The | Zinc finger protein derivatives and methods therefor |
US5877302A (en) | 1994-03-23 | 1999-03-02 | Case Western Reserve University | Compacted nucleic acids and their delivery to cells |
US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
DE69535829D1 (de) | 1994-08-20 | 2008-10-16 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von dna |
GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
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 |
JP2001500376A (ja) | 1996-09-06 | 2001-01-16 | カイロン コーポレイション | 組換えaavベクターを使用する治療分子の肝臓特異的送達のための方法および組成物 |
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 |
GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
GB9710809D0 (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 |
DE69942334D1 (de) | 1998-03-02 | 2010-06-17 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
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 |
US6824987B1 (en) | 1999-05-11 | 2004-11-30 | President And Fellows Of Harvard College | Small molecule printing |
IL150069A0 (en) | 1999-12-06 | 2002-12-01 | 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 |
IL154059A0 (en) | 2000-07-21 | 2003-07-31 | Syngenta Participations Ag | Zinc finger domain recognition code and uses thereof |
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 |
AUPR446701A0 (en) | 2001-04-18 | 2001-05-17 | Gene Stream Pty Ltd | Transgenic mammals for pharmacological and toxicological studies |
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 |
BRPI0307383B1 (pt) | 2002-01-23 | 2019-12-31 | The Univ Of Utah Research Foundation | método de recombinação genética direcionada em célula de planta hospedeira |
AU2003218382B2 (en) | 2002-03-21 | 2007-12-13 | Sangamo Therapeutics, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
JP2006502748A (ja) | 2002-09-05 | 2006-01-26 | カリフォルニア インスティテュート オブ テクノロジー | 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 |
US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
ES2315859T3 (es) | 2004-04-08 | 2009-04-01 | Sangamo Biosciences, Inc. | Metodos y composiciones para tratar afecciones neuropaticas y neurodegenerativas. |
CA2562193A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
EP2292274A1 (en) | 2004-09-16 | 2011-03-09 | Sangamo BioSciences, Inc. | Compositions and methods for protein production |
JP5017118B2 (ja) * | 2004-10-08 | 2012-09-05 | バークシス コーポレーション | 組換えタンパク質をインビボ生産するための、非常に豊富な転写産物の標的化トランススプライシング |
EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
US8043088B2 (en) | 2005-05-16 | 2011-10-25 | Johnson Douglas B | Endodontic procedure employing simultaneous liquefaction and acoustic debridgement |
KR20080033455A (ko) * | 2005-07-26 | 2008-04-16 | 상가모 바이오사이언스 인코포레이티드 | 외래 핵산 서열의 표적화된 통합 및 발현 |
ES2440801T3 (es) | 2005-10-18 | 2014-01-30 | Precision Biosciences | Meganucleasas racionalmente diseñadas con especificidad de secuencia y afinidad de unión a ADN alteradas |
AU2007267874B2 (en) | 2006-05-25 | 2012-03-15 | Sangamo Therapeutics, Inc. | Methods and compositions for gene inactivation |
DE602007005634D1 (de) | 2006-05-25 | 2010-05-12 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
JP2010500029A (ja) | 2006-08-11 | 2010-01-07 | ダウ アグロサイエンシィズ エルエルシー | ジンクフィンガーヌクレアーゼ媒介相同組換え |
JP2010516277A (ja) * | 2007-01-26 | 2010-05-20 | シナジェバ・バイオファーマ・コーポレイション | トリにおける導入遺伝子発現 |
AU2007345347B2 (en) | 2007-01-26 | 2013-11-07 | Synageva Biopharma Corp | Transgene expression in avians |
ATE489465T1 (de) | 2007-04-26 | 2010-12-15 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
CA2688834C (en) * | 2007-06-01 | 2020-01-07 | Ronald Buelow | Compositions and methods for inhibiting endogenous immunoglobulin genes and producing transgenic human idiotype antibodies |
EP2006388A1 (de) | 2007-06-19 | 2008-12-24 | Helmholtz-Zentrum für Infektionsforschung GmbH | Nukleinsäurekonstrukte zur Inaktivierung und konditionalen Mutagenese von Genen |
US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
EP2188384B1 (en) | 2007-09-27 | 2015-07-15 | Sangamo BioSciences, Inc. | Rapid in vivo identification of biologically active nucleases |
DE102007056956B4 (de) | 2007-11-27 | 2009-10-29 | Moosbauer, Peter, Dipl.-Ing.(FH) | Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung |
EP2297318B1 (en) | 2008-05-28 | 2018-05-23 | Sangamo Therapeutics, Inc. | Compositions for linking dna-binding domains and cleavage domains |
JP5908725B2 (ja) | 2008-08-22 | 2016-04-26 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 標的一本鎖開裂および標的組込みのための方法、並びに組成物 |
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 |
US20110023158A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Bovine genome editing with zinc finger nucleases |
EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
KR20120097483A (ko) * | 2009-07-24 | 2012-09-04 | 시그마-알드리치 컴퍼니., 엘엘씨 | 게놈 편집을 위한 방법 |
KR20120038020A (ko) | 2009-08-11 | 2012-04-20 | 상가모 바이오사이언스 인코포레이티드 | 표적화 변형에 대한 동형접합성 유기체 |
FR2953287B1 (fr) | 2009-11-27 | 2017-07-21 | Mc Aci | Dispositif de mesure de grandeurs parietales |
EP3456826B1 (en) * | 2009-12-10 | 2023-06-28 | Regents of the University of Minnesota | Tal effector-mediated dna modification |
WO2011097036A1 (en) | 2010-02-08 | 2011-08-11 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
EP2660318A1 (en) | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
EP3156062A1 (en) | 2010-05-17 | 2017-04-19 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
JP6018069B2 (ja) | 2010-10-12 | 2016-11-02 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 血友病bを治療する方法及び組成物 |
US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
US9394545B2 (en) * | 2011-09-21 | 2016-07-19 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
WO2014151123A1 (en) | 2013-03-15 | 2014-09-25 | Microvention, Inc. | Multi-component obstruction removal system and method |
-
2012
- 2012-09-21 US US13/624,193 patent/US9394545B2/en active Active
- 2012-09-21 AU AU2012312260A patent/AU2012312260B2/en not_active Ceased
- 2012-09-21 IL IL277027A patent/IL277027B/en unknown
- 2012-09-21 HK HK14111232.9A patent/HK1198178A1/xx unknown
- 2012-09-21 SG SG10201811736QA patent/SG10201811736QA/en unknown
- 2012-09-21 KR KR1020147010499A patent/KR102061557B1/ko active Active
- 2012-09-21 WO PCT/US2012/056539 patent/WO2013044008A2/en not_active Application Discontinuation
- 2012-09-21 IL IL293650A patent/IL293650B2/en unknown
- 2012-09-21 EP EP19154696.9A patent/EP3498833B1/en active Active
- 2012-09-21 CA CA2848417A patent/CA2848417C/en active Active
- 2012-09-21 CA CA3186126A patent/CA3186126A1/en active Pending
- 2012-09-21 SG SG11201400707TA patent/SG11201400707TA/en unknown
- 2012-09-21 EP EP12832906.7A patent/EP2758529B1/en active Active
- 2012-09-21 JP JP2014532002A patent/JP6185916B2/ja not_active Expired - Fee Related
- 2012-09-21 US US13/624,217 patent/US9150847B2/en active Active
- 2012-09-21 CN CN201810145310.0A patent/CN108610423A/zh active Pending
- 2012-09-21 CN CN201280051774.6A patent/CN103917644A/zh active Pending
- 2012-09-21 ES ES19154696T patent/ES2961613T3/es active Active
- 2012-09-21 KR KR1020197023865A patent/KR102209330B1/ko not_active Expired - Fee Related
-
2014
- 2014-03-11 IL IL231447A patent/IL231447B/en active IP Right Grant
-
2015
- 2015-08-21 US US14/832,434 patent/US9777281B2/en active Active
-
2017
- 2017-07-28 JP JP2017146622A patent/JP6496363B2/ja not_active Expired - Fee Related
- 2017-08-29 US US15/690,067 patent/US10975375B2/en not_active Expired - Fee Related
- 2017-11-20 AU AU2017264986A patent/AU2017264986B2/en not_active Ceased
-
2018
- 2018-10-09 IL IL262238A patent/IL262238B/en active IP Right Grant
- 2018-11-27 IL IL263324A patent/IL263324A/en unknown
-
2019
- 2019-03-08 JP JP2019042238A patent/JP6890145B2/ja not_active Expired - Fee Related
- 2019-04-03 US US16/374,042 patent/US11639504B2/en active Active
- 2019-07-16 AU AU2019206021A patent/AU2019206021B2/en not_active Ceased
-
2021
- 2021-03-03 US US17/190,994 patent/US11859190B2/en active Active
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
US11859190B2 (en) | Methods and compositions for regulation of transgene expression | |
JP7218401B2 (ja) | リソソーム貯蔵疾患の処置のための方法および組成物 | |
HK40009831A (en) | Methods and compositions for regulation of transgene expression | |
NZ622180B2 (en) | Methods and compositions for regulation of transgene expression | |
NZ707974B2 (en) | Methods And Compositions For Regulation Of Transgene Expression | |
NZ718174B2 (en) | Methods And Compositions For Regulation Of Transgene Expression |