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 (cg-RX-API-DMAC7.html) | 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)
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | 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 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | 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 |
| MX382223B (es) * | 2015-05-12 | 2025-03-13 | Sangamo Therapeutics Inc | Regulacion de expresion genica mediada por nucleasa. |
| KR102796744B1 (ko) | 2015-06-09 | 2025-04-15 | 에디타스 메디신, 인코포레이티드 | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 |
| GB2592821B (en) | 2015-07-31 | 2022-01-12 | Univ Minnesota | Modified cells 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 |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | 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 |
| WO2017214569A1 (en) * | 2016-06-09 | 2017-12-14 | Regents Of The University Of Minnesota | Genome-edited nk cell and methods of making and using |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| AU2017382171A1 (en) * | 2016-12-19 | 2019-07-04 | The Uab Research Foundation | Mass spectrometric standards for hemoglobin beta and hemoglobin beta sickle and uses thereof |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| AU2018212652B2 (en) * | 2017-01-26 | 2024-03-28 | Sangamo Therapeutics, Inc. | B-cell engineering |
| JP7175277B2 (ja) | 2017-01-27 | 2022-11-18 | チルドレンズ ホスピタル メディカル センター | 造血幹細胞の生着活性を向上させる方法 |
| 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 |
| US11975029B2 (en) | 2017-02-28 | 2024-05-07 | Vor Biopharma Inc. | Compositions and methods for inhibition of lineage specific proteins |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| 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 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| JP2020518245A (ja) | 2017-05-03 | 2020-06-25 | サンガモ セラピューティクス， インコーポレイテッド | 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物 |
| 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 |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| CN109722414A (zh) * | 2017-10-27 | 2019-05-07 | 博雅辑因（北京）生物科技有限公司 | 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基 |
| CA3079405A1 (en) * | 2017-10-31 | 2019-05-09 | Magenta Therapeutics Inc. | Compositions and methods for the expansion of hematopoietic stem and progenitor cells |
| JP2021502085A (ja) * | 2017-11-09 | 2021-01-28 | サンガモ セラピューティクス， インコーポレイテッド | サイトカイン誘導性ｓｈ２含有タンパク質（ｃｉｓｈ）遺伝子の遺伝子改変 |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| EP3511412A1 (en) * | 2018-01-12 | 2019-07-17 | Genethon | Genetically engineered hematopoietic stem cell as a platform for systemic protein expression |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3575402A1 (en) * | 2018-06-01 | 2019-12-04 | Algentech SAS | Gene targeting |
| SG11202101576UA (en) | 2018-08-18 | 2021-03-30 | Harvard College | In situ gene editing |
| WO2020046951A1 (en) * | 2018-08-27 | 2020-03-05 | The Regents Of The University Of California | Methods to control viral infection in mammalian cells |
| CN113423725A (zh) | 2018-08-28 | 2021-09-21 | Vor生物制药股份有限公司 | 遗传工程化造血干细胞及其用途 |
| GB201815670D0 (en) * | 2018-09-26 | 2018-11-07 | Univ Oxford Innovation Ltd | Protein editing |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | 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 |
| US20220177922A1 (en) * | 2019-03-07 | 2022-06-09 | National University Corporation Tokyo Medical And Dental University | High-throughput gene-editing technique |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| JP2022519949A (ja) | 2019-04-02 | 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 | ベータ－サラセミアの治療のための方法 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP3958879A4 (en) * | 2019-04-25 | 2023-05-10 | Actinium Pharmaceuticals, Inc. | Compositions and methods of immunodepletion for the treatment of malignant and non-malignant hematological diseases |
| EP3770264A1 (en) * | 2019-07-22 | 2021-01-27 | Genethon | Precise integration using nuclease targeted idlv |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US20240124561A1 (en) * | 2019-10-17 | 2024-04-18 | Minicircle Inc. | Nucleic acids encoding hiv neutralizing antibodies and uses thereof |
| 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 |
| 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 |
| US20240309361A1 (en) * | 2021-07-01 | 2024-09-19 | Bluebird Bio, Inc. | Methods |
| 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 |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
| WO2025194124A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | Modified st1cas9 guide nucleic acids |
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 |
| 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 |
| 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 |
| 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 |
| AU704601B2 (en) | 1994-01-18 | 1999-04-29 | Scripps Research Institute, The | Zinc finger protein derivatives and methods therefor |
| AU696455C (en) | 1994-03-23 | 2006-03-02 | 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 |
| 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 |
| ATE318327T1 (de) * | 1996-06-04 | 2006-03-15 | Univ Utah Res Found | Fluoreszenz-donor-akzeptor paar |
| 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 |
| US6479626B1 (en) | 1998-03-02 | 2002-11-12 | 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 |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| 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 |
| 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 |
| CA2398590C (en) | 2000-02-08 | 2012-08-28 | 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 |
| WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US20050267061A1 (en) | 2004-04-08 | 2005-12-01 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
| 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 |
| EP2266396A3 (en) | 2001-09-24 | 2011-06-15 | Sangamo BioSciences, Inc. | Modulation of stem cells using zinc finger proteins |
| 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 |
| EP2368982A3 (en) | 2002-03-21 | 2011-10-12 | Sangamo BioSciences, 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 |
| US9447434B2 (en) | 2002-09-05 | 2016-09-20 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
| AU2003261970A1 (en) * | 2002-09-06 | 2004-03-29 | Wakunaga Pharmaceutical Co., Ltd. | Method of identifying nucleic acid having polymorphism sequence site |
| 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 |
| CA2562193A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | 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. |
| 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 |
| ES2533370T3 (es) | 2005-10-18 | 2015-04-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| EP1999255A2 (en) | 2006-03-24 | 2008-12-10 | The Children's Medical Center Corporation | Method to modulate hematopoietic stem cell growth |
| EP2765195A1 (en) | 2006-05-25 | 2014-08-13 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| ATE462783T1 (de) | 2006-05-25 | 2010-04-15 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| 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 |
| EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | Sangamo BioSciences, Inc. | Linear donor constructs for targeted integration |
| CA2734235C (en) | 2008-08-22 | 2019-03-26 | Sangamo Biosciences, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| ES2561599T3 (es) | 2008-08-28 | 2016-02-29 | Taiga Biotechnologies, Inc. | Moduladores de MYC, métodos de uso de los mismos, y métodos para identificar agentes que modulan MYC |
| WO2010065123A1 (en) | 2008-12-04 | 2010-06-10 | Sangamo Biosciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| CA2747124C (en) | 2008-12-17 | 2018-09-04 | Dow Agrosciences Llc | Targeted integration into the zp15 locus |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| MX336846B (es) * | 2010-01-22 | 2016-02-03 | Sangamo Biosciences Inc | Alteracion genomica dirigida. |
| EP2615106B1 (en) | 2010-02-08 | 2018-04-25 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| AU2011215557B2 (en) | 2010-02-09 | 2016-03-10 | Sangamo Therapeutics, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| WO2011139336A1 (en) | 2010-04-26 | 2011-11-10 | Sangamo Biosciences, Inc. | Genome editing of a rosa locus using nucleases |
| EP2571512B1 (en) | 2010-05-17 | 2017-08-23 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
| US8945868B2 (en) | 2010-07-21 | 2015-02-03 | Sangamo Biosciences, 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 |
| EP2643454A4 (en) | 2010-11-24 | 2014-05-21 | Hong Gao | EXPANSION OF HEMATOPOETIC STEM CELLS |
| WO2013019745A1 (en) | 2011-07-29 | 2013-02-07 | Georgia Health Sciences University | Methods and compositions for genetically modifiying cells |
| IL293650B2 (en) | 2011-09-21 | 2024-10-01 | Sangamo Therapeutics Inc | Methods and compositions for regulation of transgene expression |
| AU2012328682B2 (en) | 2011-10-27 | 2017-09-21 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the HPRT locus |
| RU2018109507A (ru) | 2012-02-24 | 2019-02-28 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
| US10174331B2 (en) * | 2012-05-07 | 2019-01-08 | Sangamo Therapeutics, 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 |
| CA2878037C (en) * | 2012-07-11 | 2021-08-31 | Sangamo Biosciences, Inc. | Methods and compositions for delivery of biologics |
| CN114645015A (zh) | 2012-07-20 | 2022-06-21 | 泰加生物工艺学公司 | 造血区室的增强的重建和自动重建 |
| SG11201500852WA (en) * | 2012-08-29 | 2015-04-29 | Sangamo Biosciences Inc | Methods and compositions for treatment of a genetic condition |
| 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 |
| BR112016025849A2 (pt) | 2014-05-08 | 2017-10-17 | Chdi Foundation Inc | métodos e composições para o tratamento da doença de huntington |
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- 2015-09-16 EP EP21161850.9A patent/EP3878948A1/en not_active Withdrawn
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