CO2017012026A2 - Regulación de la expresión génica mediada por nucleasa - Google Patents
Regulación de la expresión génica mediada por nucleasaInfo
- Publication number
- CO2017012026A2 CO2017012026A2 CONC2017/0012026A CO2017012026A CO2017012026A2 CO 2017012026 A2 CO2017012026 A2 CO 2017012026A2 CO 2017012026 A CO2017012026 A CO 2017012026A CO 2017012026 A2 CO2017012026 A2 CO 2017012026A2
- Authority
- CO
- Colombia
- Prior art keywords
- regulation
- gene expression
- mediated gene
- nuclease mediated
- genome
- Prior art date
Links
- 101710163270 Nuclease Proteins 0.000 title 1
- 230000014509 gene expression Effects 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 230000004048 modification Effects 0.000 abstract 2
- 238000012986 modification Methods 0.000 abstract 2
- 101710185494 Zinc finger protein Proteins 0.000 abstract 1
- 102100023597 Zinc finger protein 816 Human genes 0.000 abstract 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- 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 RNAses, DNAses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/20—Cytokines; Chemokines
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- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Cell Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Developmental Biology & Embryology (AREA)
- Diabetes (AREA)
- Epidemiology (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)
- Peptides Or Proteins (AREA)
- Materials For Medical Uses (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
La presente descripción se encuentra en el campo de la modificación del genoma, particularmente la modificación dirigida del genoma de una célula hematopoyética usando una proteína de dedo de cinc que se une a un gen BCL11a.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562160396P | 2015-05-12 | 2015-05-12 | |
US201662303595P | 2016-03-04 | 2016-03-04 | |
PCT/US2016/032049 WO2016183298A2 (en) | 2015-05-12 | 2016-05-12 | Nuclease-mediated regulation of gene expression |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2017012026A2 true CO2017012026A2 (es) | 2018-02-28 |
Family
ID=57249092
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2017/0012026A CO2017012026A2 (es) | 2015-05-12 | 2017-11-27 | Regulación de la expresión génica mediada por nucleasa |
Country Status (17)
Country | Link |
---|---|
US (2) | US10808020B2 (es) |
EP (1) | EP3294866A4 (es) |
JP (2) | JP6873917B2 (es) |
KR (1) | KR20170141217A (es) |
AU (2) | AU2016261927B2 (es) |
BR (1) | BR112017024115A2 (es) |
CA (1) | CA2984013A1 (es) |
CL (1) | CL2017002843A1 (es) |
CO (1) | CO2017012026A2 (es) |
HK (1) | HK1251011A1 (es) |
IL (3) | IL284707B2 (es) |
MX (2) | MX2017014446A (es) |
PH (1) | PH12017502026A1 (es) |
SA (1) | SA517390307B1 (es) |
SG (1) | SG10202112057QA (es) |
WO (1) | WO2016183298A2 (es) |
ZA (1) | ZA201707016B (es) |
Families Citing this family (41)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins 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 |
EP3960856A1 (en) * | 2013-11-13 | 2022-03-02 | Children's Medical Center Corporation | Nuclease-mediated regulation of gene expression |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
EP3294866A4 (en) * | 2015-05-12 | 2018-12-05 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
CA2998500A1 (en) | 2015-09-23 | 2017-03-30 | Sangamo Therapeutics, Inc. | Htt repressors and uses thereof |
CN108699116A (zh) | 2015-10-23 | 2018-10-23 | 哈佛大学的校长及成员们 | 用于基因编辑的演化的cas9蛋白 |
WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
HUE056707T2 (hu) | 2016-08-24 | 2022-03-28 | Sangamo Therapeutics Inc | Génexpresszió szabályozása szerkezeti nukleázok alkalmazásával |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
KR20220145913A (ko) | 2016-08-24 | 2022-10-31 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
CA3039928A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
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 |
WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
EP3592777A1 (en) | 2017-03-10 | 2020-01-15 | President and Fellows of Harvard College | Cytosine to guanine base editor |
US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
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 |
JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
US11795443B2 (en) | 2017-10-16 | 2023-10-24 | The Broad Institute, Inc. | Uses of adenosine base editors |
US20200384033A1 (en) * | 2017-12-05 | 2020-12-10 | Vertex Pharmaceuticals Incorporated | Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof |
WO2019150196A1 (en) | 2018-02-05 | 2019-08-08 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
AU2019326408A1 (en) | 2018-08-23 | 2021-03-11 | Sangamo Therapeutics, Inc. | Engineered target specific base editors |
US11453639B2 (en) | 2019-01-11 | 2022-09-27 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
WO2020191243A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
EP3946384A1 (en) | 2019-04-02 | 2022-02-09 | Sangamo Therapeutics, Inc. | Methods for the treatment of beta-thalassemia |
DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
WO2022016070A1 (en) | 2020-07-16 | 2022-01-20 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
EP4255501A1 (en) * | 2020-12-01 | 2023-10-11 | Lepton Pharmaceuticals Ltd. | Methods for enhancing therapeutic efficacy of isolated cells for cell therapy |
US20230242922A1 (en) | 2021-09-13 | 2023-08-03 | Life Technologies Corporation | Gene editing tools |
WO2023081756A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
Family Cites Families (80)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
JP4012243B2 (ja) | 1994-01-18 | 2007-11-21 | ザ スクリップス リサーチ インスティチュート | 亜鉛フィンガータンパク質誘導体およびそのための方法 |
US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
USRE39229E1 (en) | 1994-08-20 | 2006-08-08 | Gendaq Limited | Binding proteins for recognition of 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 |
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 |
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 |
WO1999045132A1 (en) | 1998-03-02 | 1999-09-10 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
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 |
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 |
US7013219B2 (en) | 1999-01-12 | 2006-03-14 | 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 |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
US7030215B2 (en) * | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
EP1236045B1 (en) | 1999-12-06 | 2005-11-09 | Sangamo Biosciences Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
JP5047437B2 (ja) | 2000-02-08 | 2012-10-10 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 薬物の発見のための細胞 |
US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
US20030044787A1 (en) | 2000-05-16 | 2003-03-06 | Joung J. Keith | 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 |
EP1421177A4 (en) | 2001-08-20 | 2006-06-07 | Scripps Research Inst | ZINC FINGER FASTENING DOMAINS FOR CNN |
US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
DE60310202T2 (de) | 2002-01-23 | 2007-11-22 | The University Of Utah Research Foundation, Salt Lake City | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
AU2003218382B2 (en) | 2002-03-21 | 2007-12-13 | Sangamo Therapeutics, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
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 |
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 |
WO2005099771A2 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
AU2005233550B2 (en) | 2004-04-08 | 2010-11-18 | Sangamo Therapeutics, 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 |
MX2007013757A (es) | 2005-05-05 | 2008-01-24 | Univ Arizona | Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas. |
DE102005054628A1 (de) | 2005-11-16 | 2007-05-24 | Cevec Pharmaceuticals Gmbh | Verfahren zur Herstellung von permanenten humanen Zelllinien |
AU2007267887B2 (en) | 2006-05-25 | 2013-05-30 | Sangamo Therapeutics, Inc. | Variant Foki Cleavage Half-Domains |
EP2765195A1 (en) | 2006-05-25 | 2014-08-13 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
WO2008157688A2 (en) | 2007-06-19 | 2008-12-24 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap |
JP5763341B2 (ja) | 2007-09-27 | 2015-08-12 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法 |
CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
AU2009260888B2 (en) | 2008-05-28 | 2014-09-11 | Sangamo Therapeutics, Inc. | Compositions for linking DNA-binding domains and cleavage domains |
CN102159722B (zh) | 2008-08-22 | 2014-09-03 | 桑格摩生物科学股份有限公司 | 用于靶向单链切割和靶向整合的方法和组合物 |
MX345116B (es) | 2008-09-15 | 2017-01-17 | Children's Medical Center Corp | Modulacion de bcl11a para tratamiento de hemoglobinopatias. |
ES2627552T3 (es) | 2008-12-04 | 2017-07-28 | Sigma Aldrich Company | Edición de genoma en ratas usando nucleasas con dedos de cinc |
JP5866283B2 (ja) * | 2009-07-28 | 2016-02-17 | サンガモ バイオサイエンシーズ, インコーポレイテッド | トリヌクレオチド反復疾患を治療するための方法および組成物 |
EA022786B1 (ru) | 2009-07-31 | 2016-03-31 | Этрис Гмбх | Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков |
US8586526B2 (en) | 2010-05-17 | 2013-11-19 | Sangamo Biosciences, Inc. | DNA-binding proteins and uses thereof |
US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
ES2751916T3 (es) | 2010-02-08 | 2020-04-02 | Sangamo Therapeutics Inc | Semidominios de escisión genomanipulados |
CA2788850C (en) | 2010-02-09 | 2019-06-25 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
CN102939377B (zh) | 2010-04-26 | 2016-06-08 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶对Rosa位点进行基因组编辑 |
CA2797189C (en) | 2010-05-03 | 2020-05-26 | Sangamo Biosciences, Inc. | Compositions for linking zinc finger modules |
US8945868B2 (en) | 2010-07-21 | 2015-02-03 | Sangamo Biosciences, Inc. | Methods and compositions for modification of a HLA locus |
WO2012094132A1 (en) | 2011-01-05 | 2012-07-12 | Sangamo Biosciences, Inc. | Methods and compositions for gene correction |
EP3498833B1 (en) | 2011-09-21 | 2023-08-16 | Sangamo Therapeutics, 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 |
CA2854819C (en) | 2011-11-16 | 2022-07-19 | Sangamo Biosciences, Inc. | Modified dna-binding proteins and uses thereof |
AU2013243947A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
WO2013169802A1 (en) | 2012-05-07 | 2013-11-14 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
US9834782B2 (en) | 2012-05-28 | 2017-12-05 | Evogene Ltd. | Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics |
KR20220164090A (ko) * | 2012-08-29 | 2022-12-12 | 상가모 테라퓨틱스, 인코포레이티드 | 유전적 병태를 치료하기 위한 방법 및 조성물 |
EP2925864B1 (en) | 2012-11-27 | 2018-10-31 | The Children's Medical Center Corporation | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
CA2892448A1 (en) | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
EP3730615A3 (en) | 2013-05-15 | 2020-12-09 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
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 |
EP3960856A1 (en) | 2013-11-13 | 2022-03-02 | Children's Medical Center Corporation | Nuclease-mediated regulation of gene expression |
WO2015171932A1 (en) | 2014-05-08 | 2015-11-12 | Sangamo Biosciences, Inc. | Methods and compositions for treating huntington's disease |
ES2835861T3 (es) * | 2015-05-08 | 2021-06-23 | Childrens Medical Ct Corp | Direccionamiento de regiones funcionales del potenciador de BCL11A para la reinducción de hemoglobina fetal |
EP3294866A4 (en) * | 2015-05-12 | 2018-12-05 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
EP4053277A1 (en) | 2015-12-28 | 2022-09-07 | Novartis AG | Compositions and methods for the treatment of hemoglobinopathies |
PT3445388T (pt) * | 2016-04-18 | 2024-05-22 | Vertex Pharma | Materiais e métodos para tratamento de hemoglobinopatias |
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2016
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- 2016-05-12 BR BR112017024115A patent/BR112017024115A2/pt active Search and Examination
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- 2016-05-12 KR KR1020177032403A patent/KR20170141217A/ko not_active Application Discontinuation
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2018
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2020
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JP2021106611A (ja) | 2021-07-29 |
IL255288A0 (en) | 2017-12-31 |
JP7365374B2 (ja) | 2023-10-19 |
AU2016261927A1 (en) | 2017-11-02 |
PH12017502026A1 (en) | 2018-04-02 |
US10808020B2 (en) | 2020-10-20 |
IL287033A (en) | 2021-12-01 |
US20210024606A1 (en) | 2021-01-28 |
BR112017024115A2 (pt) | 2018-08-07 |
CL2017002843A1 (es) | 2018-05-25 |
SG10202112057QA (en) | 2021-12-30 |
HK1251011A1 (zh) | 2019-01-18 |
JP6873917B2 (ja) | 2021-05-19 |
IL255288B (en) | 2021-10-31 |
SA517390307B1 (ar) | 2021-07-13 |
AU2016261927B2 (en) | 2022-04-07 |
EP3294866A4 (en) | 2018-12-05 |
WO2016183298A3 (en) | 2016-12-29 |
MX2021005226A (es) | 2021-06-18 |
CA2984013A1 (en) | 2016-11-17 |
IL284707B2 (en) | 2024-01-01 |
EP3294866A2 (en) | 2018-03-21 |
AU2022204863A1 (en) | 2022-07-28 |
JP2018516556A (ja) | 2018-06-28 |
US20180111975A1 (en) | 2018-04-26 |
IL284707A (en) | 2021-08-31 |
WO2016183298A2 (en) | 2016-11-17 |
KR20170141217A (ko) | 2017-12-22 |
IL284707B1 (en) | 2023-09-01 |
MX2017014446A (es) | 2018-06-13 |
ZA201707016B (en) | 2019-08-28 |
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