JP2018525979A5 - - Google Patents

Download PDF

Info

Publication number
JP2018525979A5
JP2018525979A5 JP2018501179A JP2018501179A JP2018525979A5 JP 2018525979 A5 JP2018525979 A5 JP 2018525979A5 JP 2018501179 A JP2018501179 A JP 2018501179A JP 2018501179 A JP2018501179 A JP 2018501179A JP 2018525979 A5 JP2018525979 A5 JP 2018525979A5
Authority
JP
Japan
Prior art keywords
cell
composition
nuclease
donor molecule
introducing
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.)
Granted
Application number
JP2018501179A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018525979A (ja
JP6954890B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/042024 external-priority patent/WO2017011519A1/en
Publication of JP2018525979A publication Critical patent/JP2018525979A/ja
Publication of JP2018525979A5 publication Critical patent/JP2018525979A5/ja
Priority to JP2020209010A priority Critical patent/JP7348160B2/ja
Application granted granted Critical
Publication of JP6954890B2 publication Critical patent/JP6954890B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018501179A 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 Active JP6954890B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2020209010A JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562191918P 2015-07-13 2015-07-13
US62/191,918 2015-07-13
US201562247469P 2015-10-28 2015-10-28
US62/247,469 2015-10-28
US201662315438P 2016-03-30 2016-03-30
US62/315,438 2016-03-30
PCT/US2016/042024 WO2017011519A1 (en) 2015-07-13 2016-07-13 Delivery methods and compositions for nuclease-mediated genome engineering

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020209010A Division JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Publications (3)

Publication Number Publication Date
JP2018525979A JP2018525979A (ja) 2018-09-13
JP2018525979A5 true JP2018525979A5 (cg-RX-API-DMAC7.html) 2019-08-22
JP6954890B2 JP6954890B2 (ja) 2021-10-27

Family

ID=57758305

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018501179A Active JP6954890B2 (ja) 2015-07-13 2016-07-13 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2020209010A Active JP7348160B2 (ja) 2015-07-13 2020-12-17 ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物

Country Status (7)

Country Link
US (2) US10450585B2 (cg-RX-API-DMAC7.html)
EP (1) EP3322297B1 (cg-RX-API-DMAC7.html)
JP (2) JP6954890B2 (cg-RX-API-DMAC7.html)
CN (1) CN108024544B (cg-RX-API-DMAC7.html)
AU (1) AU2016291778B2 (cg-RX-API-DMAC7.html)
CA (1) CA2991301A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017011519A1 (cg-RX-API-DMAC7.html)

Families Citing this family (115)

* Cited by examiner, † Cited by third party
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
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9752113B2 (en) 2012-03-15 2017-09-05 Flodesign Sonics, Inc. Acoustic perfusion devices
US10322949B2 (en) 2012-03-15 2019-06-18 Flodesign Sonics, Inc. Transducer and reflector configurations for an acoustophoretic device
US10689609B2 (en) 2012-03-15 2020-06-23 Flodesign Sonics, Inc. Acoustic bioreactor processes
US9745548B2 (en) 2012-03-15 2017-08-29 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10737953B2 (en) 2012-04-20 2020-08-11 Flodesign Sonics, Inc. Acoustophoretic method for use in bioreactors
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9745569B2 (en) 2013-09-13 2017-08-29 Flodesign Sonics, Inc. System for generating high concentration factors for low cell density suspensions
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3092049A1 (en) 2014-01-08 2016-11-16 Flodesign Sonics Inc. Acoustophoresis device with dual acoustophoretic chamber
US9744483B2 (en) 2014-07-02 2017-08-29 Flodesign Sonics, Inc. Large scale acoustic separation device
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
US11667911B2 (en) 2015-09-24 2023-06-06 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/CAS-mediated genome editing
EP3940070A1 (en) 2015-10-05 2022-01-19 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
KR20250013291A (ko) 2015-10-05 2025-01-31 프리시젼 바이오사이언시스 인코포레이티드 변형된 인간 t 세포 수용체 알파 불변 영역 유전자를 포함하는 유전자 변형된 세포
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
EP4628587A2 (en) 2016-04-15 2025-10-08 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
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
CA3034882A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
BR112019003327A2 (pt) * 2016-08-24 2019-07-02 Sangamo Therapeutics, Inc. nucleases manipuladas de alvo específico
AU2017338827B2 (en) 2016-10-03 2023-08-31 Juno Therapeutics, Inc. HPV-specific binding molecules
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
KR20190127655A (ko) 2016-10-19 2019-11-13 프로디자인 소닉스, 인크. 음향학에 의한 친화성 세포 추출
US20200087681A1 (en) * 2016-12-21 2020-03-19 Ucl Business Plc Therapeutic cells
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
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 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
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
MX2019015188A (es) 2017-06-15 2020-08-03 Univ California Inserciones de adn no virales orientadas.
EP3638783A4 (en) * 2017-06-16 2021-03-10 Sangamo Therapeutics, Inc. TARGETED T-LYMPHOCYTE AND / OR HLA RECEPTOR INTERRUPTION
CN111511375A (zh) * 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
CA3068465A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
WO2019006330A1 (en) * 2017-06-30 2019-01-03 Indiana University Research And Technology Corporation COMPOSITIONS AND METHODS FOR DETECTING REACTIVITY TO ALS
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
CN111194350A (zh) * 2017-09-18 2020-05-22 博雅辑因(北京)生物科技有限公司 一种基因编辑t细胞及其用途
IL273427B2 (en) * 2017-09-22 2025-04-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous polycythemia vera type II
US11952408B2 (en) 2017-10-03 2024-04-09 Juno Therapeutics, Inc. HPV-specific binding molecules
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
MX2020004325A (es) 2017-10-27 2020-11-09 Univ California Reemplazo dirigido de receptores de células t endógenos.
AU2018383712B2 (en) * 2017-12-11 2025-09-04 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
JP2021507561A (ja) 2017-12-14 2021-02-22 フロデザイン ソニックス, インク.Flodesign Sonics, Inc. 音響トランスデューサドライバ及びコントローラ
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
US20210015869A1 (en) 2018-04-05 2021-01-21 Juno Therapeutics, Inc. T cells expressing a recombinant receptor, related polynucleotides and methods
CN112585276A (zh) 2018-04-05 2021-03-30 朱诺治疗学股份有限公司 产生表达重组受体的细胞的方法和相关组合物
RU2020135968A (ru) 2018-04-05 2022-05-06 Джуно Терапьютикс, Инк. T-клеточные рецепторы и модифицированные клетки, экспрессирующие
IL277883B2 (en) 2018-04-12 2023-12-01 Prec Biosciences Inc Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
AU2019296451B2 (en) 2018-06-29 2021-04-29 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
MX2021001375A (es) 2018-08-03 2021-04-19 Sangamo Therapeutics Inc Parametros clinicos mejorados por la expresion del factor viii.
US20200063160A1 (en) * 2018-08-07 2020-02-27 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type ii
CN108949763A (zh) * 2018-08-15 2018-12-07 吉林大学 能有效抑制猪瘟病毒感染的精确突变LamR基因及应用
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3840785A4 (en) 2018-08-20 2022-07-13 Wuhan Neurophth Biotechnology Limited Company COMPOSITIONS AND METHODS FOR THE TREATMENT OF LEBERIAN OPTIC ATROPHY
WO2020038352A1 (en) * 2018-08-20 2020-02-27 Wuhan Neurophth Biological Technology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CA3120093A1 (en) 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Compositions and methods for compartment-specific cargo delivery
WO2020102503A2 (en) * 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3826664A4 (en) 2019-02-06 2022-10-26 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type i
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220016475A (ko) 2019-05-01 2022-02-09 주노 쎄러퓨티크스 인코퍼레이티드 변형된 tgfbr2 유전자 좌에서 재조합 수용체를 발현하는 세포, 관련 폴리뉴클레오티드 및 방법
EP3962519A1 (en) 2019-05-01 2022-03-09 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods
US11622547B2 (en) 2019-06-07 2023-04-11 Regeneran Pharmaceuticals, Inc. Genetically modified mouse that expresses human albumin
EP4004052A4 (en) * 2019-07-30 2023-11-01 Provention Bio, Inc. METHODS AND COMPOSITIONS FOR REDUCING IMMUNOGENICITY BY NON-DEPLETED B CELL INHIBITORS
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN113025633B (zh) 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
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
KR20230022868A (ko) 2020-05-13 2023-02-16 주노 쎄러퓨티크스 인코퍼레이티드 재조합 수용체를 발현하는 공여자-배치 세포의 제조 방법
WO2021260186A1 (en) 2020-06-26 2021-12-30 Juno Therapeutics Gmbh Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
US20230398148A1 (en) 2020-11-04 2023-12-14 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods
WO2023064928A2 (en) 2021-10-14 2023-04-20 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
WO2023081900A1 (en) 2021-11-08 2023-05-11 Juno Therapeutics, Inc. Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods
WO2023122036A1 (en) * 2021-12-20 2023-06-29 Sobol Robert E Methods and compositions for improved molecular therapies of multigenic diseases
EP4615960A1 (en) 2022-11-09 2025-09-17 C3S2 GmbH Methods for manufacturing engineered immune cells
EP4658675A1 (en) 2023-02-03 2025-12-10 C3S2 GmbH Methods for non-viral manufacturing of engineered immune cells
WO2024229281A1 (en) * 2023-05-02 2024-11-07 City Of Hope Adeno-associated virus compositions and methods of use thereof
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117858A1 (en) * 2023-11-28 2025-06-05 Metagenomi, Inc. Supplementation of liver enzyme expression
WO2025212519A1 (en) 2024-04-01 2025-10-09 Moonlight Bio, Inc. Dll3 binding proteins and uses thereof
WO2025235851A1 (en) 2024-05-10 2025-11-13 Juno Therapeutics, Inc. Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US255250A (en) 1882-03-21 Whistling toy
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
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
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (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
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
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
US6479626B1 (en) 1998-03-02 2002-11-12 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140815A (en) 1998-06-17 2000-10-31 Dover Instrument Corporation High stability spin stand platform
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
NZ511379A (en) * 1998-11-10 2003-06-30 Ludwig Inst Cancer Res Platelet-derived growth factor D (PDGF-D), DNA coding therefor, and uses thereof for invasions of tumor cells
US7013219B2 (en) 1999-01-12 2006-03-14 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
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
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
US6978476B2 (en) 2000-05-16 2005-12-20 Comsonics Device and method of determining location of signal ingress
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
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20030003583A1 (en) 2001-06-08 2003-01-02 Raphael Hirsch Regulation of transgene expression following AAV transduction
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute 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
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
GB0312086D0 (en) * 2003-05-27 2003-07-02 Novartis Ag Organic compounds
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
WO2005113003A2 (en) * 2004-04-16 2005-12-01 Genentech, Inc. Method for augmenting b cell depletion
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.
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
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
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
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
KR101297192B1 (ko) 2008-11-10 2013-08-19 삼성전자주식회사 화상형성장치, 칩, 및, 칩 패키지
EP2359134B1 (en) 2008-11-17 2014-06-04 Koninklijke Nederlandse Akademie van Wetenschappen Methods for identifying modulating compounds of lymphangiogenesis, means therefore, compounds and uses thereof
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
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
JP2013523776A (ja) * 2010-04-02 2013-06-17 セレジーン インコーポレイテッド パーキンソン病および脳のドーパミン作動性ニューロンのその他の障害を処置する方法
WO2011139336A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using nucleases
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
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
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
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
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
CN102633883B (zh) * 2012-02-24 2014-06-25 上海白泽生物科技有限公司 一种能与egfr、her2、vegf高效结合的融合蛋白、其编码序列及用途
US10174331B2 (en) 2012-05-07 2019-01-08 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
EP2855666B1 (en) * 2012-05-25 2019-12-04 Cellectis Use of pre t alpha or functional variant thereof for expanding tcr alpha deficient t cells
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
CA2886684C (en) * 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP2929017A4 (en) 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES
US10272163B2 (en) * 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
US9999206B2 (en) * 2013-07-25 2018-06-19 Utah State University Model and method for a transgenic bovidae expressing cardiac fibrosis and associated pathology
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
WO2015164758A1 (en) * 2014-04-25 2015-10-29 University Of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
BR112016025849A2 (pt) 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
KR20250013291A (ko) * 2015-10-05 2025-01-31 프리시젼 바이오사이언시스 인코포레이티드 변형된 인간 t 세포 수용체 알파 불변 영역 유전자를 포함하는 유전자 변형된 세포
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof

Similar Documents

Publication Publication Date Title
JP2018525979A5 (cg-RX-API-DMAC7.html)
JP7348160B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物
Mangeot et al. Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins
JP6818720B2 (ja) カスパーゼポリペプチドを使用して部分的なアポトーシスを誘導するための方法
Yin et al. Delivery technologies for genome editing
US11186825B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting POU2AF1
US10280402B2 (en) Immune-compatible cells created by nuclease-mediated editing of genes encoding HLA
US11180730B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting GATA3
JP6722176B2 (ja) 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
JP2024001104A (ja) 改変された細胞および治療の方法
US20190100801A1 (en) Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
JP2022531231A (ja) Cd8改変t細胞療法を使用するがんの治療のための組成物及び方法
JP7428712B2 (ja) 低/最小操作による遺伝子改変細胞の製造
JP2018171075A (ja) 改変されたカスパーゼポリペプチドおよびその使用
Rosigkeit et al. Monitoring translation activity of mRNA-loaded nanoparticles in mice
EP3314020A1 (en) Tumor and microenvironment gene expression, compositions of matter and methods of use thereof
Meissner et al. Immune editing: overcoming immune barriers in stem cell transplantation
JP2016539627A5 (cg-RX-API-DMAC7.html)
KR102741572B1 (ko) 조직에서 코딩 리보핵산의 표적화된 전달, 발현 및 조절을 위한 조성물 및 방법
WO2017132291A1 (en) Genetic, developmental and micro-environmental programs in idh-mutant gliomas, compositions of matter and methods of use thereof
WO2022256448A2 (en) Compositions and methods for targeting, editing, or modifying genes
Zirger et al. Immune-mediated loss of transgene expression from virally transduced brain cells is irreversible, mediated by IFNγ, perforin, and TNFα, and due to the elimination of transduced cells
JP2023546300A (ja) 細胞治療のための方法および組成物
Zheng et al. Engineering of human mesenchymal stem cells resistant to multiple natural killer subtypes
JP2025518552A (ja) 細胞を操作するための組成物及び方法