PL3504229T3 - Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii - Google Patents

Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii

Info

Publication number
PL3504229T3
PL3504229T3 PL17844404T PL17844404T PL3504229T3 PL 3504229 T3 PL3504229 T3 PL 3504229T3 PL 17844404 T PL17844404 T PL 17844404T PL 17844404 T PL17844404 T PL 17844404T PL 3504229 T3 PL3504229 T3 PL 3504229T3
Authority
PL
Poland
Prior art keywords
regulation
gene expression
engineered nucleases
nucleases
engineered
Prior art date
Application number
PL17844404T
Other languages
English (en)
Inventor
Jeffrey C. Miller
Edward J. Rebar
Original Assignee
Sangamo Therapeutics, Inc.
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sangamo Therapeutics, Inc. filed Critical Sangamo Therapeutics, Inc.
Publication of PL3504229T3 publication Critical patent/PL3504229T3/pl

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
PL17844404T 2016-08-24 2017-08-24 Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii PL3504229T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662378978P 2016-08-24 2016-08-24
US201762443981P 2017-01-09 2017-01-09
US201762545778P 2017-08-15 2017-08-15
EP17844404.8A EP3504229B1 (en) 2016-08-24 2017-08-24 Regulation of gene expression using engineered nucleases
PCT/US2017/048397 WO2018039440A1 (en) 2016-08-24 2017-08-24 Regulation of gene expression using engineered nucleases

Publications (1)

Publication Number Publication Date
PL3504229T3 true PL3504229T3 (pl) 2022-04-19

Family

ID=61246246

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17844404T PL3504229T3 (pl) 2016-08-24 2017-08-24 Regulacja ekspresji genów przy użyciu nukleaz poddanych inżynierii

Country Status (32)

Country Link
US (2) US10563184B2 (pl)
EP (2) EP3504229B1 (pl)
JP (1) JP7203014B2 (pl)
KR (1) KR102572759B1 (pl)
CN (1) CN110325635B (pl)
AU (2) AU2017315406B2 (pl)
BR (1) BR112019003100A2 (pl)
CA (1) CA3034882A1 (pl)
CL (1) CL2019000399A1 (pl)
CO (1) CO2019001364A2 (pl)
CR (1) CR20190098A (pl)
DK (1) DK3504229T3 (pl)
DO (1) DOP2019000031A (pl)
EC (1) ECSP19017082A (pl)
ES (1) ES2900317T3 (pl)
HR (1) HRP20212025T1 (pl)
HU (1) HUE056707T2 (pl)
IL (1) IL264639B2 (pl)
LT (1) LT3504229T (pl)
MX (1) MX2019002207A (pl)
MY (1) MY193234A (pl)
NZ (1) NZ750938A (pl)
PE (1) PE20190568A1 (pl)
PH (1) PH12019500281A1 (pl)
PL (1) PL3504229T3 (pl)
PT (1) PT3504229T (pl)
RS (1) RS62758B1 (pl)
SG (2) SG11201901531TA (pl)
SI (1) SI3504229T1 (pl)
TN (1) TN2019000048A1 (pl)
WO (1) WO2018039440A1 (pl)
ZA (1) ZA201900765B (pl)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 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 ヌクレアーゼ切断特異性の評価および改善
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
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
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
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
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
WO2018039448A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Engineered target specific nucleases
LT3504229T (lt) * 2016-08-24 2021-12-10 Sangamo Therapeutics, Inc. Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
KR20190085529A (ko) * 2016-12-01 2019-07-18 상가모 테라퓨틱스, 인코포레이티드 Tau 조절제 및 그것의 전달을 위한 방법 및 조성물
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20190130613A (ko) 2017-03-23 2019-11-22 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
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (en) 2017-08-30 2019-07-18 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
WO2019157324A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
KR20210143230A (ko) 2019-03-19 2021-11-26 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물
EP3946384A1 (en) 2019-04-02 2022-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
CN116096873A (zh) 2020-05-08 2023-05-09 布罗德研究所股份有限公司 同时编辑靶标双链核苷酸序列的两条链的方法和组合物
CN114149990A (zh) * 2020-09-08 2022-03-08 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
US11514747B2 (en) 2020-09-30 2022-11-29 Aristocrat Technologies, Inc. Latching assembly for a pushbutton of an electronic gaming machine
WO2023014839A1 (en) * 2021-08-03 2023-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of sickle cell disease

Family Cites Families (123)

* Cited by examiner, † Cited by third party
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
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
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
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
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
EP0770129B1 (en) 1994-01-18 2005-11-23 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
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
WO1995025809A1 (en) 1994-03-23 1995-09-28 Ohio University Compacted nucleic acids and their delivery to cells
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
AU698152B2 (en) 1994-08-20 1998-10-22 Gendaq Limited Improvements in or relating to 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
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
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
JP4309051B2 (ja) 1998-03-02 2009-08-05 マサチューセッツ インスティテュート オブ テクノロジー 改善したリンカーを有するポリジンクフィンガータンパク質
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
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
IL150069A0 (en) 1999-12-06 2002-12-01 Sangamo Biosciences Inc Methods of using randomized libraries of zinc finger proteins for the identification of gene function
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
WO2002057294A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger proteins for dna binding and gene regulation in plants
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
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
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
EP1732614B1 (en) 2004-04-08 2008-12-24 Sangamo Biosciences Inc. Compositions for treating neuropathic and neurodegenerative conditions
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
WO2006121866A2 (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
WO2007139982A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
AU2007284748B2 (en) 2006-08-11 2013-05-16 Corteva Agriscience Llc Zinc finger nuclease-mediated homologous recombination
CA2684378C (en) 2007-04-26 2016-11-29 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
US8585526B2 (en) 2007-06-18 2013-11-19 American Axle & Manufacturing, Inc. Vehicle driveline component having heat sink for increased heat rejection capabilities
US8153773B2 (en) 2007-06-19 2012-04-10 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger RNA cap
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
JP2011500082A (ja) 2007-10-25 2011-01-06 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方法および組成物
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
JP2011521643A (ja) 2008-05-28 2011-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド Ｄｎａ結合ドメインおよび切断ドメインを連結するための組成物
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
PT2334794T (pt) 2008-09-15 2017-02-15 Harvard College Modulação de bcl11a para tratamento de hemoglobinopatias
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
CN105255881A (zh) 2009-07-31 2016-01-20 埃泽瑞斯公司 用于蛋白质表达的具有未修饰和修饰核苷酸的组合的rna
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
BR112012012444A2 (pt) 2009-11-27 2015-09-22 Basf Plant Science Co Gmbh "endonuclease quimérica, polinucleotídeo isolado, cassete de expressão, vetor, organismo não humano, método para fornecer uma endonuclease quimérica, método para recombinação homóloga de polinucleotídeos, método para mutação alvejada de polinucleotídeosa e método para recombinação homóloga ou mutação alvejada"
IN2012DN06629A (pl) 2010-01-28 2015-10-23 Philadelphia Children Hospital
JP6137596B2 (ja) 2010-02-08 2017-05-31 サンガモ セラピューティクス， インコーポレイテッド 遺伝子操作された切断ハーフドメイン
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
EP2558571A4 (en) * 2010-04-16 2014-09-24 Immune Disease Inst Inc DELAYED POLYPEPTIDE EXPRESSION FROM MODIFIED SYNTHETIC RNAS AND USES THEREOF
CN102939377B (zh) 2010-04-26 2016-06-08 桑格摩生物科学股份有限公司 使用锌指核酸酶对Rosa位点进行基因组编辑
LT2566972T (lt) 2010-05-03 2020-03-10 Sangamo Therapeutics, Inc. Kompozicijos, skirtos cinko pirštu modulių susiejimui
CN103025344B (zh) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 新型dna-结合蛋白及其用途
US8945868B2 (en) 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
US9057057B2 (en) 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
CN102358902B (zh) * 2011-04-02 2013-01-02 西南大学 家蚕丝素重链基因突变序列及突变的方法和应用
CN103917644A (zh) 2011-09-21 2014-07-09 桑格摩生物科学股份有限公司 调控转基因表达的方法和组合物
AU2012328682B2 (en) 2011-10-27 2017-09-21 Sangamo Therapeutics, Inc. Methods and compositions for modification of the HPRT locus
EP2780460B1 (en) 2011-11-16 2018-07-11 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
CN103361328B (zh) * 2012-03-30 2017-08-18 复旦大学 介导整合的hiv‑1前病毒基因敲除的锌指核酸内切酶及其制法和应用
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
SG11201407802WA (en) 2012-05-25 2015-01-29 Cellectis Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy
SI2890780T1 (sl) * 2012-08-29 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje genetskega stanja
JP2016500254A (ja) 2012-12-05 2016-01-12 サンガモ バイオサイエンシーズ， インコーポレイテッド 代謝疾患の調節のための方法および組成物
US10030063B2 (en) * 2012-12-18 2018-07-24 Novartis Ag Production of therapeutic proteins in genetically modified mammalian cells
EP3865586A1 (en) 2013-03-15 2021-08-18 The General Hospital Corporation Increasing specificity for rna-guided genome editing
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
RS62559B1 (sr) 2013-11-13 2021-12-31 Childrens Medical Center Nukleazom posredovana regulacija eksprimiranja gena
WO2015089077A2 (en) * 2013-12-09 2015-06-18 Sangamo Biosciences, Inc. Methods and compositions for genome engineering
CN107405411A (zh) * 2014-05-01 2017-11-28 华盛顿大学 使用腺病毒载体的体内基因改造
KR102380324B1 (ko) 2014-05-08 2022-03-30 상가모 테라퓨틱스, 인코포레이티드 헌팅턴병을 치료하기 위한 방법 및 조성물
WO2016014794A1 (en) * 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
EP3054007A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step
BR112017024115A2 (pt) 2015-05-12 2018-08-07 Sangamo Therapeutics Inc regulação de expressão genética mediada por nuclease
US9957501B2 (en) * 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
WO2018039448A1 (en) 2016-08-24 2018-03-01 Sangamo Therapeutics, Inc. Engineered target specific nucleases
LT3504229T (lt) * 2016-08-24 2021-12-10 Sangamo Therapeutics, Inc. Genų raiškos reguliavimas, panaudojant rekombinantines nukleazes
US20180185516A1 (en) 2016-12-09 2018-07-05 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
AU2018313167A1 (en) 2017-08-08 2020-02-27 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting

Also Published As

Publication number Publication date
PH12019500281A1 (en) 2019-08-05
CA3034882A1 (en) 2018-03-01
US11845965B2 (en) 2023-12-19
EP3504229A4 (en) 2020-02-12
CN110325635B (zh) 2023-12-26
HRP20212025T1 (hr) 2022-04-01
US20190177709A1 (en) 2019-06-13
IL264639A (pl) 2019-03-31
WO2018039440A1 (en) 2018-03-01
PT3504229T (pt) 2021-12-13
KR20190039530A (ko) 2019-04-12
ZA201900765B (en) 2023-02-22
IL264639B1 (en) 2023-09-01
KR102572759B1 (ko) 2023-08-29
EP3995574A1 (en) 2022-05-11
BR112019003100A2 (pt) 2019-07-09
LT3504229T (lt) 2021-12-10
AU2021204432B2 (en) 2022-12-01
CO2019001364A2 (es) 2019-03-18
JP2019524150A (ja) 2019-09-05
CL2019000399A1 (es) 2019-04-26
TN2019000048A1 (en) 2020-07-15
CN110325635A (zh) 2019-10-11
EP3504229B1 (en) 2021-10-13
AU2017315406B2 (en) 2021-04-01
ES2900317T3 (es) 2022-03-16
IL264639B2 (en) 2024-01-01
US10563184B2 (en) 2020-02-18
RS62758B1 (sr) 2022-01-31
SG10201913315SA (en) 2020-02-27
SG11201901531TA (en) 2019-03-28
JP7203014B2 (ja) 2023-01-12
DK3504229T3 (da) 2021-12-20
SI3504229T1 (sl) 2022-04-29
HUE056707T2 (hu) 2022-03-28
PE20190568A1 (es) 2019-04-22
AU2021204432A1 (en) 2021-07-29
CR20190098A (es) 2019-04-30
DOP2019000031A (es) 2019-09-30
US20200131490A1 (en) 2020-04-30
MY193234A (en) 2022-09-27
MX2019002207A (es) 2019-07-15
AU2017315406A1 (en) 2019-03-14
ECSP19017082A (es) 2019-03-29
NZ750938A (en) 2023-07-28
EP3504229A1 (en) 2019-07-03

Similar Documents

Publication Publication Date Title
ZA201900765B (en) Regulation of gene expression using engineered nucleases
IL287033A (en) Regulation of nuclease-mediated gene expression
IL284796B (en) Control of gene expression through aptamer-mediated modulation of alternative splicing
HK1257676A1 (zh) 工程化 crispr-cas 9 核酸酶
SG10202007392SA (en) Engineered target specific nucleases
HUE044540T2 (hu) Nukleáz közvetítette génexpresszió-szabályozás
HK1244032A1 (zh) 控制基因表達的天然型mirna及其用途
IL257500A (en) Altered cullin1 gene
GB201705121D0 (en) Modulation of gene expression