JP2018516556A5 - - Google Patents
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- JP2018516556A5 JP2018516556A5 JP2017559044A JP2017559044A JP2018516556A5 JP 2018516556 A5 JP2018516556 A5 JP 2018516556A5 JP 2017559044 A JP2017559044 A JP 2017559044A JP 2017559044 A JP2017559044 A JP 2017559044A JP 2018516556 A5 JP2018516556 A5 JP 2018516556A5
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- Prior art keywords
- seq
- cell
- protein
- composition
- fusion protein
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 210000004027 cell Anatomy 0.000 claims description 63
- 108020001507 fusion proteins Proteins 0.000 claims description 20
- 102000037865 fusion proteins Human genes 0.000 claims description 20
- 102000004169 proteins and genes Human genes 0.000 claims description 20
- 108090000623 proteins and genes Proteins 0.000 claims description 20
- 238000000034 method Methods 0.000 claims description 15
- 108091033319 polynucleotide Proteins 0.000 claims description 14
- 102000040430 polynucleotide Human genes 0.000 claims description 14
- 239000002157 polynucleotide Substances 0.000 claims description 14
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 12
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 10
- 101710185494 Zinc finger protein Proteins 0.000 claims description 8
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 8
- 238000010362 genome editing Methods 0.000 claims description 8
- 210000000130 stem cell Anatomy 0.000 claims description 7
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 claims description 6
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims description 6
- 238000003776 cleavage reaction Methods 0.000 claims description 4
- 238000012217 deletion Methods 0.000 claims description 4
- 230000037430 deletion Effects 0.000 claims description 4
- 208000034737 hemoglobinopathy Diseases 0.000 claims description 4
- 230000007017 scission Effects 0.000 claims description 4
- 238000013518 transcription Methods 0.000 claims description 4
- 230000035897 transcription Effects 0.000 claims description 4
- 230000002159 abnormal effect Effects 0.000 claims description 3
- 238000012986 modification Methods 0.000 claims description 3
- 230000004048 modification Effects 0.000 claims description 3
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 2
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 2
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims description 2
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 238000010322 bone marrow transplantation Methods 0.000 claims description 2
- 102000018146 globin Human genes 0.000 claims description 2
- 108060003196 globin Proteins 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 208000007056 sickle cell anemia Diseases 0.000 claims description 2
- 230000004936 stimulating effect Effects 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 13
- 208000018337 inherited hemoglobinopathy Diseases 0.000 claims 1
- 230000035800 maturation Effects 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021071881A JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562160396P | 2015-05-12 | 2015-05-12 | |
| US62/160,396 | 2015-05-12 | ||
| US201662303595P | 2016-03-04 | 2016-03-04 | |
| US62/303,595 | 2016-03-04 | ||
| PCT/US2016/032049 WO2016183298A2 (en) | 2015-05-12 | 2016-05-12 | Nuclease-mediated regulation of gene expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021071881A Division JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018516556A JP2018516556A (ja) | 2018-06-28 |
| JP2018516556A5 true JP2018516556A5 (OSRAM) | 2019-06-13 |
| JP6873917B2 JP6873917B2 (ja) | 2021-05-19 |
Family
ID=57249092
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017559044A Active JP6873917B2 (ja) | 2015-05-12 | 2016-05-12 | ヌクレアーゼ介在性遺伝子発現調節 |
| JP2021071881A Active JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021071881A Active JP7365374B2 (ja) | 2015-05-12 | 2021-04-21 | ヌクレアーゼ介在性遺伝子発現調節 |
Country Status (17)
| Country | Link |
|---|---|
| US (2) | US10808020B2 (OSRAM) |
| EP (1) | EP3294866A4 (OSRAM) |
| JP (2) | JP6873917B2 (OSRAM) |
| KR (1) | KR20170141217A (OSRAM) |
| AU (2) | AU2016261927B2 (OSRAM) |
| BR (1) | BR112017024115A2 (OSRAM) |
| CA (1) | CA2984013A1 (OSRAM) |
| CL (1) | CL2017002843A1 (OSRAM) |
| CO (1) | CO2017012026A2 (OSRAM) |
| HK (1) | HK1251011A1 (OSRAM) |
| IL (3) | IL284707B2 (OSRAM) |
| MX (2) | MX382223B (OSRAM) |
| PH (1) | PH12017502026A1 (OSRAM) |
| SA (1) | SA517390307B1 (OSRAM) |
| SG (1) | SG10202112057QA (OSRAM) |
| WO (1) | WO2016183298A2 (OSRAM) |
| ZA (1) | ZA201707016B (OSRAM) |
Families Citing this family (54)
| 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 |
| 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 |
| ES2895069T3 (es) | 2013-11-13 | 2022-02-17 | Childrens Medical Center | Regulación de la expresión génica mediada por nucleasa |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| BR112017024115A2 (pt) * | 2015-05-12 | 2018-08-07 | Sangamo Therapeutics Inc | regulação de expressão genética mediada por nuclease |
| US10435441B2 (en) | 2015-09-23 | 2019-10-08 | Sangamo Therapeutics, Inc. | HTT repressors and uses thereof |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| IL264792B2 (en) | 2016-08-24 | 2023-10-01 | Sangamo Therapeutics Inc | Engineered target-specific zinc-finger 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 |
| AU2017315406B2 (en) | 2016-08-24 | 2021-04-01 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | 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 | 核酸塩基エディターのａａｖ送達 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | 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 |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| 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 |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | 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 |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| MA51107A (fr) * | 2017-12-05 | 2020-10-14 | Vertex Pharma | Cellules souches et progénitrices hématopoïétiques humaines cd34+ modifiées par crispr-cas9 et utilisations associées |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3749767A1 (en) | 2018-02-05 | 2020-12-16 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12486519B2 (en) | 2018-08-18 | 2025-12-02 | President And Fellows Of Harvard College | In situ gene editing |
| US11834686B2 (en) | 2018-08-23 | 2023-12-05 | Sangamo Therapeutics, Inc. | Engineered target specific base editors |
| IL281615B1 (en) | 2018-09-21 | 2025-09-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| AU2020205717B2 (en) | 2019-01-11 | 2025-09-11 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| MA55531A (fr) | 2019-04-02 | 2022-02-09 | Sangamo Therapeutics Inc | Procédés pour le traitement de béta-thalassémie |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| 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 |
| CA3189338A1 (en) | 2020-07-16 | 2022-01-20 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
| AU2021392094A1 (en) * | 2020-12-01 | 2023-07-06 | Lepton Pharmaceuticals Ltd. | Methods for enhancing therapeutic efficacy of isolated cells for cell therapy |
| KR20240055084A (ko) | 2021-09-13 | 2024-04-26 | 라이프 테크놀로지스 코포레이션 | 유전자 편집 도구 |
| CA3237482A1 (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 |
| WO2023114943A2 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| 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 |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (114)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPH08510134A (ja) * | 1993-05-17 | 1996-10-29 | 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 | Ｈｉｖ感染およびエイズを対象としたリボザイム遺伝子治療 |
| 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 |
| CA2681922C (en) | 1994-01-18 | 2012-05-15 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| JP4118327B2 (ja) | 1994-08-20 | 2008-07-16 | ゲンダック・リミテッド | Ｄｎａ認識のための結合タンパク質におけるまたはそれに関連する改良 |
| 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 |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| 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 |
| ATE466952T1 (de) | 1998-03-02 | 2010-05-15 | 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 |
| 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 |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| 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 |
| DE60023936T2 (de) | 1999-12-06 | 2006-05-24 | Sangamo Biosciences Inc., Richmond | Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen |
| US6689558B2 (en) | 2000-02-08 | 2004-02-10 | 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 |
| 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 |
| US20040224385A1 (en) | 2001-08-20 | 2004-11-11 | Barbas Carlos F | Zinc finger binding 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 |
| EP1476547B1 (en) * | 2002-01-23 | 2006-12-06 | The University of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| 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 |
| EP1581610A4 (en) | 2002-09-05 | 2009-05-27 | California Inst Of Techn | USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING |
| EP3222715A1 (en) * | 2003-08-08 | 2017-09-27 | 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 |
| 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 |
| AU2005233550B2 (en) | 2004-04-08 | 2010-11-18 | Sangamo Therapeutics, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| DE602005011943D1 (de) | 2004-04-08 | 2009-02-05 | Sangamo Biosciences Inc | Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen |
| US20060063231A1 (en) | 2004-09-16 | 2006-03-23 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| 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 |
| US20100055793A1 (en) * | 2005-07-25 | 2010-03-04 | Johns Hopkins University | Site-specific modification of the human genome using custom-designed zinc finger nucleases |
| WO2007014275A2 (en) * | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| DE102005054628A1 (de) * | 2005-11-16 | 2007-05-24 | Cevec Pharmaceuticals Gmbh | Verfahren zur Herstellung von permanenten humanen Zelllinien |
| AU2007254251A1 (en) * | 2006-05-19 | 2007-11-29 | Sangamo Therapeutics, Inc. | Methods and compositions for inactivation of dihydrofolate reductase |
| JP5266210B2 (ja) | 2006-05-25 | 2013-08-21 | サンガモ バイオサイエンシズ インコーポレイテッド | 改変開裂ハーフドメイン |
| WO2007139982A2 (en) | 2006-05-25 | 2007-12-06 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
| US9428756B2 (en) * | 2006-08-11 | 2016-08-30 | Dow Agrosciences Llc | Zinc finger nuclease-mediated homologous recombination |
| DE602008003684D1 (de) * | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| CN104072561B (zh) | 2007-06-19 | 2017-12-22 | 路易斯安那州州立大学及农业机械学院管理委员会 | 信使rna帽的抗‑反向硫代磷酸类似物的合成和用途 |
| WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| AU2008305590A1 (en) * | 2007-09-27 | 2009-04-02 | Sangamo Therapeutics, Inc. | Genomic editing in zebrafish using zinc finger nucleases |
| US9506120B2 (en) | 2007-09-27 | 2016-11-29 | Sangamo Biosciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | Sangamo BioSciences, Inc. | Linear donor constructs for targeted integration |
| EP2297318B1 (en) | 2008-05-28 | 2018-05-23 | Sangamo Therapeutics, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| CA2734235C (en) | 2008-08-22 | 2019-03-26 | Sangamo Biosciences, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| EP3208339B1 (en) | 2008-09-15 | 2019-05-01 | The Children's Medical Center Corporation | Modulation of bcl11a for treatment of hemoglobinopathies |
| US8153399B2 (en) * | 2008-10-29 | 2012-04-10 | Sangamo Biosciences, Inc. | Methods and compositions for inactivating glutamine synthetase gene expression |
| US9206404B2 (en) | 2008-12-04 | 2015-12-08 | Sangamo Biosciences, Inc. | Method of deleting an IgM gene in an isolated rat cell |
| AU2010235161B2 (en) * | 2009-04-09 | 2015-01-22 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
| WO2011016840A2 (en) * | 2009-07-28 | 2011-02-10 | Sangamo Biosciences, Inc. | Methods and compositions for treating trinucleotide repeat disorders |
| EP3165234B1 (de) | 2009-07-31 | 2019-04-03 | ethris GmbH | Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression |
| KR101813981B1 (ko) * | 2009-10-22 | 2018-01-04 | 다우 아그로사이언시즈 엘엘씨 | 지방산 생합성에 포함된 식물 유전자를 표적하는 조작된 징크 핑거 단백질 |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| JP5902631B2 (ja) * | 2010-01-22 | 2016-04-13 | ダウ アグロサイエンシィズ エルエルシー | 標的化ゲノム変更 |
| CA2788560A1 (en) * | 2010-02-08 | 2011-08-11 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| WO2011100058A1 (en) * | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| EP2563918B1 (en) | 2010-04-26 | 2016-11-23 | Sangamo BioSciences, Inc. | Genome editing of a rosa locus using zinc-finger nucleases |
| CN102959087B (zh) | 2010-05-03 | 2015-04-29 | 桑格摩生物科学股份有限公司 | 用于连接锌指模块的组合物 |
| CN103025344B (zh) | 2010-05-17 | 2016-06-29 | 桑格摩生物科学股份有限公司 | 新型dna-结合蛋白及其用途 |
| US8945868B2 (en) | 2010-07-21 | 2015-02-03 | Sangamo Biosciences, Inc. | Methods and compositions for modification of a HLA locus |
| KR20180121665A (ko) * | 2010-07-23 | 2018-11-07 | 시그마-알드리치 컴퍼니., 엘엘씨 | 표적화 엔도뉴클레아제 및 단일-가닥 핵산을 사용하는 게놈 편집 |
| US20140127814A1 (en) * | 2010-08-10 | 2014-05-08 | Srinivasan Chandrasegaran | Generation and use of pluripotent stem cells |
| WO2012087756A1 (en) * | 2010-12-22 | 2012-06-28 | Sangamo Biosciences, Inc. | Zinc finger nuclease modification of leucine rich repeat kinase 2 (lrrk2) mutant fibroblasts and ipscs |
| KR101556359B1 (ko) * | 2011-01-03 | 2015-10-01 | 주식회사 툴젠 | 디자인된 tal 이펙터 뉴클레아제를 통한 게놈 엔지니어링 |
| US9267123B2 (en) | 2011-01-05 | 2016-02-23 | Sangamo Biosciences, Inc. | Methods and compositions for gene correction |
| WO2012106725A2 (en) * | 2011-02-04 | 2012-08-09 | Sangamo Biosciences, Inc. | Methods and compositions for treating occular disorders |
| EP2522726A1 (en) * | 2011-05-12 | 2012-11-14 | Fundació Privada Centre de Regulació Genòmica (CRG) | Zinc finger nucleases for p53 editing |
| WO2012152912A1 (en) * | 2011-05-12 | 2012-11-15 | Newvectys | Genetically modified pig as a cancer prone model |
| US9161995B2 (en) * | 2011-07-25 | 2015-10-20 | Sangamo Biosciences, Inc. | Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene |
| WO2013019745A1 (en) * | 2011-07-29 | 2013-02-07 | Georgia Health Sciences University | Methods and compositions for genetically modifiying cells |
| 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 |
| BR112014014105B1 (pt) * | 2011-12-16 | 2023-10-10 | Targetgene Biotechnologies Ltd | Método para modificar um sítio-alvo predeterminado no interior de um ácido nucleico genômico ou organelar alvo em uma célula hospedeira eucariótica por um complexo núcleoproteína |
| US20130216579A1 (en) * | 2012-01-23 | 2013-08-22 | The Trustees Of Columbia University In The City Of New York | Methods and compostitions for gene editing of a pathogen |
| SG10201606959PA (en) * | 2012-02-24 | 2016-09-29 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| AU2013243952A1 (en) * | 2012-04-02 | 2014-10-30 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| WO2013158309A2 (en) * | 2012-04-18 | 2013-10-24 | The Board Of Trustees Of The Leland Stanford Junior University | Non-disruptive gene targeting |
| CA2871524C (en) | 2012-05-07 | 2021-07-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| DK3473707T3 (da) * | 2012-05-25 | 2025-03-17 | Cellectis | Fremgangsmåder til engineering af allogen og immunsuppressiv resistent t-celle til immunoterapi |
| BR112014029908B1 (pt) * | 2012-05-28 | 2022-02-15 | Evogene Ltd | Método para aumentar a produção, taxa de crescimento, biomassa, vigor, e/ou eficiência no uso de nitrogênio de uma planta, e, construção de ácido nucléico |
| SG11201500852WA (en) | 2012-08-29 | 2015-04-29 | Sangamo Biosciences Inc | Methods and compositions for treatment of a genetic condition |
| EP2925864B1 (en) * | 2012-11-27 | 2018-10-31 | The Children's Medical Center Corporation | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
| EP2929017A4 (en) | 2012-12-05 | 2016-09-28 | Sangamo Biosciences Inc | METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES |
| EP3138911B1 (en) * | 2012-12-06 | 2018-12-05 | Sigma Aldrich Co. LLC | Crispr-based genome modification and regulation |
| US10272163B2 (en) * | 2012-12-07 | 2019-04-30 | The Regents Of The University Of California | Factor VIII mutation repair and tolerance induction |
| HK1223401A1 (zh) * | 2013-05-15 | 2017-07-28 | 桑格摩生物科学股份有限公司 | 用於治疗遗传病状的方法和组合物 |
| 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 |
| EP3057432B1 (en) * | 2013-10-17 | 2018-11-21 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
| CN105916983A (zh) * | 2013-10-25 | 2016-08-31 | 塞勒克提斯公司 | 用于高效且特异性靶向包含高度重复基序的dna序列的稀有切割核酸内切酶的设计 |
| ES2895069T3 (es) * | 2013-11-13 | 2022-02-17 | Childrens Medical Center | Regulación de la expresión génica mediada por nucleasa |
| AU2015255877B2 (en) | 2014-05-08 | 2020-03-26 | Chdi Foundation, Inc. | Methods and compositions for treating huntington's disease |
| AU2015317793B2 (en) * | 2014-09-16 | 2021-11-11 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |
| AU2016225179C1 (en) * | 2015-02-23 | 2022-11-03 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| US11572543B2 (en) | 2015-05-08 | 2023-02-07 | The Children's Medical Center. Corporation | Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction |
| BR112017024115A2 (pt) * | 2015-05-12 | 2018-08-07 | Sangamo Therapeutics Inc | regulação de expressão genética mediada por nuclease |
| WO2017077394A2 (en) * | 2015-11-04 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| NZ783532A (en) | 2015-12-28 | 2025-09-26 | Novartis Ag | Compositions and methods for the treatment of hemoglobinopathies |
| MX2018012729A (es) * | 2016-04-18 | 2019-07-04 | Crispr Therapeutics Ag | Materiales y metodos para el tratamiento de hemoglobinopatias. |
| WO2017191503A1 (en) * | 2016-05-05 | 2017-11-09 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
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- 2016-05-12 KR KR1020177032403A patent/KR20170141217A/ko not_active Ceased
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