JP2017532955A5 - - Google Patents
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- JP2017532955A5 JP2017532955A5 JP2017514521A JP2017514521A JP2017532955A5 JP 2017532955 A5 JP2017532955 A5 JP 2017532955A5 JP 2017514521 A JP2017514521 A JP 2017514521A JP 2017514521 A JP2017514521 A JP 2017514521A JP 2017532955 A5 JP2017532955 A5 JP 2017532955A5
- Authority
- JP
- Japan
- Prior art keywords
- cell
- genetically modified
- zinc finger
- modified cell
- item
- 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 56
- 101150013707 HBB gene Proteins 0.000 claims description 16
- 238000000034 method Methods 0.000 claims description 16
- 108091033319 polynucleotide Proteins 0.000 claims description 16
- 102000040430 polynucleotide Human genes 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 210000003743 erythrocyte Anatomy 0.000 claims description 12
- 108020001507 fusion proteins Proteins 0.000 claims description 10
- 102000037865 fusion proteins Human genes 0.000 claims description 10
- 230000014509 gene expression Effects 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 101710163270 Nuclease Proteins 0.000 claims description 6
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 6
- 101710185494 Zinc finger protein Proteins 0.000 claims description 6
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 6
- 238000010362 genome editing Methods 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 5
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 4
- 238000003780 insertion Methods 0.000 claims description 4
- 230000037431 insertion Effects 0.000 claims description 4
- 238000004519 manufacturing process Methods 0.000 claims description 4
- 230000035772 mutation Effects 0.000 claims description 4
- 239000002243 precursor Substances 0.000 claims description 4
- 239000011701 zinc Substances 0.000 claims description 4
- 229910052725 zinc Inorganic materials 0.000 claims description 4
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 2
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 2
- 108091034117 Oligonucleotide Proteins 0.000 claims description 2
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000012239 gene modification Methods 0.000 claims description 2
- 230000005017 genetic modification Effects 0.000 claims description 2
- 235000013617 genetically modified food Nutrition 0.000 claims description 2
- 238000010348 incorporation Methods 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 238000012545 processing Methods 0.000 claims description 2
- 230000007017 scission Effects 0.000 claims description 2
- 230000004936 stimulating effect Effects 0.000 claims description 2
- 238000000338 in vitro Methods 0.000 claims 2
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019150566A JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462051159P | 2014-09-16 | 2014-09-16 | |
| US62/051,159 | 2014-09-16 | ||
| PCT/US2015/050411 WO2016044416A1 (en) | 2014-09-16 | 2015-09-16 | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019150566A Division JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017532955A JP2017532955A (ja) | 2017-11-09 |
| JP2017532955A5 true JP2017532955A5 (enExample) | 2018-08-16 |
| JP6722176B2 JP6722176B2 (ja) | 2020-07-15 |
Family
ID=55533790
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017514521A Expired - Fee Related JP6722176B2 (ja) | 2014-09-16 | 2015-09-16 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2019150566A Expired - Fee Related JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2021128240A Pending JP2021182931A (ja) | 2014-09-16 | 2021-08-04 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019150566A Expired - Fee Related JP6929585B2 (ja) | 2014-09-16 | 2019-08-20 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
| JP2021128240A Pending JP2021182931A (ja) | 2014-09-16 | 2021-08-04 | 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物 |
Country Status (18)
| Country | Link |
|---|---|
| US (1) | US10435677B2 (enExample) |
| EP (2) | EP3878948A1 (enExample) |
| JP (3) | JP6722176B2 (enExample) |
| KR (1) | KR20170054445A (enExample) |
| CN (2) | CN113699113A (enExample) |
| AU (2) | AU2015317793B2 (enExample) |
| CA (1) | CA2960769A1 (enExample) |
| DK (1) | DK3194570T3 (enExample) |
| ES (1) | ES2886012T3 (enExample) |
| HR (1) | HRP20211468T1 (enExample) |
| HU (1) | HUE055583T2 (enExample) |
| LT (1) | LT3194570T (enExample) |
| PL (1) | PL3194570T3 (enExample) |
| PT (1) | PT3194570T (enExample) |
| RS (1) | RS62334B1 (enExample) |
| SG (2) | SG10202011572XA (enExample) |
| SI (1) | SI3194570T1 (enExample) |
| WO (1) | WO2016044416A1 (enExample) |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP7030522B2 (ja) * | 2015-05-11 | 2022-03-07 | エディタス・メディシン、インコーポレイテッド | 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法 |
| HK1251011A1 (zh) * | 2015-05-12 | 2019-01-18 | Sangamo Therapeutics, Inc. | 核酸酶介导的基因表达调控 |
| JP7396783B2 (ja) | 2015-06-09 | 2023-12-12 | エディタス・メディシン、インコーポレイテッド | 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物 |
| WO2017023801A1 (en) | 2015-07-31 | 2017-02-09 | Regents Of The University Of Minnesota | Intracellular genomic transplant and methods of therapy |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| US20200208111A1 (en) * | 2016-06-09 | 2020-07-02 | Branden S. Moriarity | Genome-edited nk cell and methods of making and using |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| JP2020507059A (ja) * | 2016-12-19 | 2020-03-05 | ザ ユーエービー リサーチ ファンデーション | ヘモグロビンβ及び鎌状赤血球ヘモグロビンβのための質量分析スタンダードならびにその使用 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| BR112019015355A2 (pt) * | 2017-01-26 | 2020-05-19 | Sangamo Therapeutics Inc | modificação genética de células b |
| CA3050514A1 (en) | 2017-01-27 | 2018-08-02 | Children's Hospital Medical Center | Methods of enhancing engraftment activity of hematopoietic stem cells |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| WO2018160768A1 (en) | 2017-02-28 | 2018-09-07 | Vor Biopharma, Inc. | Compositions and methods for inhibition lineage specific proteins |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| RU2019139045A (ru) * | 2017-05-03 | 2021-06-03 | Сангамо Терапьютикс, Инк. | Способы и композиции для модификации гена регулятора трансмембранной проводимости при кистозном фиброзе (cftr) |
| 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 |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| CN109722415B (zh) * | 2017-10-27 | 2021-01-26 | 博雅辑因（北京）生物科技有限公司 | 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法 |
| US20190314407A1 (en) * | 2017-10-31 | 2019-10-17 | Magenta Therapeutics Inc. | Compositions and methods for the expansion of hematopoietic stem and progenitor cells |
| SG11202004003YA (en) * | 2017-11-09 | 2020-05-28 | Sangamo Therapeutics Inc | Genetic modification of cytokine inducible sh2-containing protein (cish) gene |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3511412A1 (en) * | 2018-01-12 | 2019-07-17 | Genethon | Genetically engineered hematopoietic stem cell as a platform for systemic protein expression |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3575402A1 (en) * | 2018-06-01 | 2019-12-04 | Algentech SAS | Gene targeting |
| KR20210058816A (ko) | 2018-08-18 | 2021-05-24 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 원 위치에 유전자 편집 |
| US20210340501A1 (en) * | 2018-08-27 | 2021-11-04 | The Regents Of The University Of California | Methods to Control Viral Infection in Mammalian Cells |
| MX2021002415A (es) | 2018-08-28 | 2021-09-21 | Vor Biopharma Inc | Celulas madre hematopoyeticas modificadas geneticamente y usos de las mismas. |
| GB201815670D0 (en) * | 2018-09-26 | 2018-11-07 | Univ Oxford Innovation Ltd | Protein editing |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7699357B2 (ja) * | 2019-03-07 | 2025-06-27 | 国立大学法人東京科学大学 | ハイスループット遺伝子編集技術 |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| PH12021552499A1 (en) | 2019-04-02 | 2022-09-19 | Sangamo Therapeutics Inc | Methods for the treatment of beta-thalassemia |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| JP2022530100A (ja) * | 2019-04-25 | 2022-06-27 | アクティニウム ファーマシューティカルズ インコーポレイテッド | 悪性および非悪性血液疾患の治療のための免疫枯渇の組成物および方法 |
| EP3770264A1 (en) * | 2019-07-22 | 2021-01-27 | Genethon | Precise integration using nuclease targeted idlv |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| WO2021076965A1 (en) * | 2019-10-17 | 2021-04-22 | Minicircle, Inc. | Nucleic acids encoding hiv neutralizing antibodies and uses thereof |
| GB2614813B (en) | 2020-05-08 | 2025-05-07 | Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2022094363A1 (en) * | 2020-11-01 | 2022-05-05 | University of South Alabama Foundation for Research and Commercialization | Barcoded cells engineered with heterozygous genetic diversity |
| CN115232811B (zh) * | 2021-06-28 | 2025-10-28 | 南京启真基因工程有限公司 | 用于构建hbb基因突变的镰刀型细胞贫血症模型猪核移植供体细胞的方法及应用 |
| JP2024524465A (ja) * | 2021-07-01 | 2024-07-05 | ブルーバード バイオ， インコーポレイテッド | 方法 |
| 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 |
| WO2025194124A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | Modified st1cas9 guide nucleic acids |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
Family Cites Families (108)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| 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 |
| 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 |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| 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 |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| 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 |
| 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 |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
| DE69535853D1 (de) | 1994-03-23 | 2008-11-20 | Univ Case Western Reserve | Kompaktnukleinsäure und ihre verabreichung in zellen |
| US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| USRE39229E1 (en) | 1994-08-20 | 2006-08-08 | Gendaq Limited | Binding proteins for recognition of DNA |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| WO1997046714A1 (en) * | 1996-06-04 | 1997-12-11 | University Of Utah Research Foundation | Monitoring hybridization during pcr |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| US6342345B1 (en) | 1997-04-02 | 2002-01-29 | The Board Of Trustees Of The Leland Stanford Junior University | Detection of molecular interactions by reporter subunit complementation |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| 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 |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6503717B2 (en) | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| 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 |
| 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 |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| 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 |
| US7030846B2 (en) | 2001-07-10 | 2006-04-18 | Samsung Electronics Co., Ltd. | Color correction liquid crystal display and method of driving same |
| WO2003016496A2 (en) | 2001-08-20 | 2003-02-27 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| EP1435779A4 (en) | 2001-09-24 | 2005-06-01 | Sangamo Biosciences Inc | MODULATION OF STEM CELLS USING ZINC FINGER PROTEINS (ZFP) |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| JP4968498B2 (ja) | 2002-01-23 | 2012-07-04 | ユニバーシティ オブ ユタ リサーチ ファウンデーション | ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発 |
| 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 |
| EP2806025B1 (en) | 2002-09-05 | 2019-04-03 | California Institute of Technology | Use of zinc finger nucleases to stimulate gene targeting |
| CN100471952C (zh) * | 2002-09-06 | 2009-03-25 | 湧永制药株式会社 | 具有多态性序列位点的核酸的鉴定方法 |
| 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 |
| CA2561714A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
| US7253273B2 (en) | 2004-04-08 | 2007-08-07 | 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 |
| CA2607104A1 (en) | 2005-05-05 | 2006-11-16 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| JP5139271B2 (ja) | 2005-05-24 | 2013-02-06 | ホワイトヘッド インスティチュート フォー バイオメディカル リサーチ | 培養した造血幹細胞を拡大しかつ分析する方法 |
| SG10201508995QA (en) | 2005-07-26 | 2015-11-27 | Sangamo Biosciences Inc | Targeted integration and expression of exogenous nucleic acid sequences |
| US8021867B2 (en) | 2005-10-18 | 2011-09-20 | Duke University | Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity |
| EP3424507A1 (en) | 2006-03-24 | 2019-01-09 | Children's Medical Center Corporation | Method to modulate hematopoietic stem cell growth |
| WO2007139898A2 (en) | 2006-05-25 | 2007-12-06 | Sangamo Biosciences, Inc. | Variant foki cleavage half-domains |
| JP5551432B2 (ja) | 2006-05-25 | 2014-07-16 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 遺伝子不活性化のための方法と組成物 |
| CN101679977B (zh) | 2007-04-26 | 2013-05-01 | 桑格摩生物科学股份有限公司 | 向ppp1r12c基因座的靶向整合 |
| US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
| US8563314B2 (en) | 2007-09-27 | 2013-10-22 | Sangamo Biosciences, Inc. | Methods and compositions for modulating PD1 |
| JP2011518555A (ja) | 2008-04-14 | 2011-06-30 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 標的組込みのための線形ドナーコンストラクト |
| EP2313515B1 (en) | 2008-08-22 | 2015-03-04 | Sangamo BioSciences, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| CN104353066B (zh) | 2008-08-28 | 2017-05-10 | 泰加生物工艺学公司 | Myc的调节剂、其使用方法和鉴别调节myc的试剂的方法 |
| AU2009322964B2 (en) | 2008-12-04 | 2014-10-09 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| AU2009333863B2 (en) | 2008-12-17 | 2015-02-05 | Corteva Agriscience Llc | Targeted integration into the Zp15 locus |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| 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 |
| EA031322B1 (ru) * | 2010-01-22 | 2018-12-28 | Дау Агросайенсиз Ллс | Клетка или клеточная линия для экспрессии экзогенных нуклеотидных последовательностей и применение клетки или клеточной линии |
| CA2788560A1 (en) | 2010-02-08 | 2011-08-11 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| CA2788850C (en) | 2010-02-09 | 2019-06-25 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| KR101880536B1 (ko) | 2010-04-26 | 2018-07-23 | 상가모 테라퓨틱스, 인코포레이티드 | 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집 |
| EP2596011B1 (en) | 2010-07-21 | 2018-10-03 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a hla locus |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| WO2012071513A2 (en) | 2010-11-24 | 2012-05-31 | Hong Gao | Expanding hematopoietic stem cells |
| WO2013019745A1 (en) | 2011-07-29 | 2013-02-07 | Georgia Health Sciences University | Methods and compositions for genetically modifiying cells |
| CA3186126A1 (en) | 2011-09-21 | 2013-03-28 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| JP6188703B2 (ja) | 2011-10-27 | 2017-08-30 | サンガモ セラピューティクス， インコーポレイテッド | Ｈｐｒｔ遺伝子座を修飾するための方法および組成物 |
| RU2018109507A (ru) | 2012-02-24 | 2019-02-28 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
| CA2871524C (en) * | 2012-05-07 | 2021-07-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| WO2013169398A2 (en) * | 2012-05-09 | 2013-11-14 | Georgia Tech Research Corporation | Systems and methods for improving nuclease specificity and activity |
| EP2872154B1 (en) * | 2012-07-11 | 2017-05-31 | Sangamo BioSciences, Inc. | Methods and compositions for delivery of biologics |
| DK2877189T3 (da) | 2012-07-20 | 2021-02-22 | Taiga Biotechnologies Inc | Forbedret rekonstituering og autorekonstituering af det hæmopoietiske kammer |
| RS60838B1 (sr) * | 2012-08-29 | 2020-10-30 | Sangamo Therapeutics Inc | Postupci i kompozicije za tretman genskog stanja |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| WO2014186585A2 (en) | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| EP3139966B1 (en) | 2014-05-08 | 2020-11-18 | Sangamo Therapeutics, Inc. | Compositions for use in treating huntington's disease |
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