IN2015DN01480A - - Google Patents

Info

Publication number
IN2015DN01480A
IN2015DN01480A IN1480DEN2015A IN2015DN01480A IN 2015DN01480 A IN2015DN01480 A IN 2015DN01480A IN 1480DEN2015 A IN1480DEN2015 A IN 1480DEN2015A IN 2015DN01480 A IN2015DN01480 A IN 2015DN01480A
Authority
IN
India
Prior art keywords
compositions
methods
genetic disease
genetic
disease
Prior art date
Application number
Other languages
English (en)
Inventor
Gregory J Cost
Philip D Gregory
Dmitry Guschin
Michael C Holmes
Jeffrey C Miller
David Paschon
Edward J Rebar
Andreas Reik
Fyodor Urnov
Lei Zhang
Original Assignee
Sangamo Biosciences 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 Biosciences Inc filed Critical Sangamo Biosciences Inc
Publication of IN2015DN01480A publication Critical patent/IN2015DN01480A/en

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • C12N2510/02—Cells for production

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
IN1480DEN2015 2012-08-29 2013-08-29 IN2015DN01480A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261694693P 2012-08-29 2012-08-29
PCT/US2013/057214 WO2014036219A2 (en) 2012-08-29 2013-08-29 Methods and compositions for treatment of a genetic condition

Publications (1)

Publication Number Publication Date
IN2015DN01480A true IN2015DN01480A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html) 2015-07-03

Family

ID=50184630

Family Applications (1)

Application Number Title Priority Date Filing Date
IN1480DEN2015 IN2015DN01480A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html) 2012-08-29 2013-08-29

Country Status (26)

Country Link
US (4) US9650648B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
EP (1) EP2890780B8 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
JP (4) JP2015533786A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
KR (3) KR102218562B1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
CN (1) CN104704110B (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
AU (1) AU2013308770B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
CA (2) CA2882499C (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
CL (1) CL2015000450A1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
DK (1) DK2890780T3 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
EA (1) EA039384B1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
ES (1) ES2812599T3 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
HR (1) HRP20201443T1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
HU (1) HUE050516T2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
IL (1) IL237050B (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
IN (1) IN2015DN01480A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
LT (1) LT2890780T (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
MX (1) MX367081B (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
PH (3) PH12021551661A1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
PL (1) PL2890780T3 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
PT (1) PT2890780T (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
RS (1) RS60838B1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
SG (2) SG10201701601WA (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
SI (1) SI2890780T1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
UA (1) UA118957C2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
WO (1) WO2014036219A2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
ZA (1) ZA201500900B (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)

Families Citing this family (122)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
RU2650811C2 (ru) 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
SI3597749T1 (sl) 2012-05-25 2023-11-30 The Regents Of The University Of California Metode in sestavki za modifikacijo ciljne dnk, usmerjene v rnk, in za modulacijo transkripcije, usmerjene v rnk
EP4357457B1 (en) * 2012-10-23 2024-10-16 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
DK3138910T3 (en) 2012-12-06 2017-10-16 Sigma Aldrich Co Llc CRISPR-BASED RE-MODIFICATION AND REGULATION
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
EP2971167B1 (en) 2013-03-14 2019-07-31 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
CN105518146B (zh) 2013-04-04 2022-07-15 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
EP4389903A3 (en) * 2013-06-05 2024-10-16 Duke University Rna-guided gene editing and gene regulation
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CA2920899C (en) * 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
AU2014346559B2 (en) 2013-11-07 2020-07-09 Editas Medicine,Inc. CRISPR-related methods and compositions with governing gRNAs
WO2015073683A2 (en) * 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
PL3102673T3 (pl) * 2014-02-03 2020-11-02 Sangamo Therapeutics, Inc. Sposoby i kompozycje do leczenia talasemii beta
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3119878B1 (en) 2014-03-18 2021-05-26 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
EP3981876A1 (en) * 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
EP3122181A4 (en) * 2014-03-26 2018-04-25 The Brigham and Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
US9970030B2 (en) 2014-08-27 2018-05-15 Caribou Biosciences, Inc. Methods for increasing CAS9-mediated engineering efficiency
JP6722176B2 (ja) * 2014-09-16 2020-07-15 サンガモ セラピューティクス， インコーポレイテッド 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
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
CN107429231A (zh) * 2014-11-24 2017-12-01 儿童医学中心公司 调节sh2b3以改善来自干细胞和/或祖细胞的红细胞的产生
WO2016094880A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
AU2016225178B2 (en) * 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2016135559A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
EP3910641B1 (en) 2015-04-13 2022-10-05 Invitae Corporation Methods, systems and processes of identifying genetic variation in highly similar genes
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
WO2016183041A2 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods
CA2984013A1 (en) * 2015-05-12 2016-11-17 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
EP3325018B1 (en) * 2015-07-22 2025-01-15 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
CA2998500A1 (en) * 2015-09-23 2017-03-30 Sangamo Therapeutics, Inc. Htt repressors and uses thereof
EP3365440B1 (en) * 2015-10-20 2022-09-14 Pioneer Hi-Bred International, Inc. Restoring function to a non-functional gene product via guided cas systems and methods of use
US10167457B2 (en) 2015-10-23 2019-01-01 President And Fellows Of Harvard College Nucleobase editors and uses thereof
CA3002524A1 (en) 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof
EP3371306B8 (en) * 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN108699542A (zh) 2015-11-13 2018-10-23 阿维利诺美国实验室股份有限公司 用于治疗角膜营养不良的方法
CN108699132B (zh) 2015-12-18 2023-08-11 桑格摩生物治疗股份有限公司 Mhc细胞受体的靶向破坏
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
IL261714B2 (en) * 2016-03-14 2024-10-01 Editas Medicine Inc Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
CN109963945A (zh) 2016-08-20 2019-07-02 阿维利诺美国实验室股份有限公司 单一向导rna、crispr/cas9系统及其使用方法
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
PE20190568A1 (es) * 2016-08-24 2019-04-22 Sangamo Therapeutics Inc Regulacion de la expresion genica usando nucleasas modificadas
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
NZ750943A (en) 2016-08-24 2025-06-27 Sangamo Therapeutics Inc Engineered target specific nucleases
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2018071663A1 (en) * 2016-10-14 2018-04-19 Emendobio Inc. Rna compositions for genome editing
CA3039928A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TWI758316B (zh) * 2017-01-09 2022-03-21 美商聖加莫治療股份有限公司 使用經工程改造之核酸酶調控基因表現
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
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN110914426A (zh) 2017-03-23 2020-03-24 哈佛大学的校长及成员们 包含核酸可编程dna结合蛋白的核碱基编辑器
US12110499B2 (en) 2017-04-24 2024-10-08 Seattle Children's Hospital Homology directed repair compositions for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
CA3070242A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC 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
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
US11661611B2 (en) * 2017-11-09 2023-05-30 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible SH2-containing protein (CISH) gene
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
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749350A4 (en) * 2018-02-08 2021-12-01 Sangamo Therapeutics, Inc. SPECIFICALLY MODIFIED TARGET-SPECIFIC NUCLEASES
EP3746153A4 (en) 2018-02-21 2021-11-10 Alcyone Lifesciences, Inc. FLUID DISTRIBUTION SYSTEMS AND METHODS
BR112020018364A2 (pt) 2018-03-14 2020-12-29 Editas Medicine, Inc. Sistemas e métodos para o tratamento de hemoglobinopatias
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CA3110345A1 (en) 2018-08-27 2020-03-05 Alcyone Lifesciences, Inc. Fluid delivery systems and methods
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
EP3946384A1 (en) 2019-04-02 2022-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
US20220184137A1 (en) * 2019-04-12 2022-06-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Correction of Beta-Thalassemia Phenotype by Genetically Engineered Hematopoietic Stem Cell
CN112391410B (zh) * 2020-02-17 2024-04-02 华东师范大学 一种sgRNA及其在修复内含子异常剪接中的应用
WO2021022189A1 (en) * 2019-08-01 2021-02-04 The Regents Of The University Of California Compositions and methods for treating alpha thalassemia
EP4010004A4 (en) * 2019-08-07 2023-09-13 Altius Institute For Biomedical Sciences Compositions and methods for modulation of gene expression
JP2022548399A (ja) * 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法
US20230227513A1 (en) * 2019-10-15 2023-07-20 Altius Institute For Biomedical Sciences DNA Binding Proteins for Displacing Endogenous Transcription Factors Bound to Gene Regulatory Regions
WO2021216622A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
US20230165909A1 (en) 2020-04-21 2023-06-01 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
WO2023115039A2 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023150518A1 (en) 2022-02-01 2023-08-10 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
EP4590688A1 (en) 2022-09-21 2025-07-30 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
EP4602174A1 (en) 2022-10-13 2025-08-20 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
WO2024243340A1 (en) 2023-05-23 2024-11-28 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2025054202A1 (en) 2023-09-05 2025-03-13 Sana Biotechnology, Inc. Method of screening a sample comprising a transgene with a unique barcode
WO2025151838A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use

Family Cites Families (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
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
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
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
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (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
EP0770129B1 (en) 1994-01-18 2005-11-23 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69535853D1 (de) 1994-03-23 2008-11-20 Univ Case Western Reserve Kompaktnukleinsäure und ihre verabreichung in zellen
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE45795E1 (en) 1994-08-20 2015-11-10 Gendaq, Ltd. 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
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
CA2321938C (en) 1998-03-02 2009-11-24 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
CO5070700A1 (es) 1998-06-05 2001-08-28 Basf Ag Genes novedosos de (adp-ribosa) polimerasa
US6140815A (en) 1998-06-17 2000-10-31 Dover Instrument Corporation High stability spin stand platform
US6532231B1 (en) 1998-08-29 2003-03-11 Lucent Technologies Inc. Arrangement for changing the destination of single link, single destination data messages
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
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
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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
WO2000046386A2 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
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
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
EP2207032A1 (en) 2000-02-08 2010-07-14 Sangamo BioSciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
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
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
EP1504092B2 (en) 2002-03-21 2014-06-25 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
EP1581610A4 (en) 2002-09-05 2009-05-27 California Inst Of Techn USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING
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
EP1651660B1 (en) 2003-08-08 2018-01-24 Sangamo Therapeutics, 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
WO2006033859A2 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
US7358085B2 (en) 2005-02-28 2008-04-15 Sangamo Biosciences, Inc. Anti-angiogenic methods and compositions
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
ES2829549T3 (es) 2005-10-18 2021-06-01 Prec Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
ES2378333T3 (es) 2006-05-25 2012-04-11 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación de genes
EP2137310B1 (en) 2007-04-26 2010-11-24 Sangamo BioSciences, Inc. Targeted integration into the ppp1r12c locus
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
AU2008305567B2 (en) 2007-09-27 2014-04-10 Sangamo Therapeutics, Inc. Rapid in vivo identification of biologically active nucleases
AU2008317354B2 (en) 2007-10-25 2014-04-10 Ospedale San Raffaele S.R.L. Methods and compositions for targeted integration
BE1018531A4 (nl) 2007-10-31 2011-03-01 Gielen Cornelis Het gebruik van niet-pathogene bacterien in sporenvorm of in slapende vorm voor het verwijderen van huismijtuitwerpselen en organische allergieverwekkende materialen van alle soorten textiel.
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
WO2009131632A1 (en) 2008-04-14 2009-10-29 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
WO2009154686A1 (en) 2008-05-28 2009-12-23 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
CN102159722B (zh) 2008-08-22 2014-09-03 桑格摩生物科学股份有限公司 用于靶向单链切割和靶向整合的方法和组合物
EP3208339B1 (en) * 2008-09-15 2019-05-01 The Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
SG172760A1 (en) 2008-12-04 2011-08-29 Sangamo Biosciences Inc Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
KR20120038020A (ko) 2009-08-11 2012-04-20 상가모 바이오사이언스 인코포레이티드 표적화 변형에 대한 동형접합성 유기체
KR102110608B1 (ko) 2009-12-10 2020-05-14 리전츠 오브 더 유니버스티 오브 미네소타 Ｔａｌ 이펙터-매개된 ｄｎａ 변형
CA2787494C (en) 2010-01-22 2019-09-17 Dow Agrosciences Llc Targeted genomic alteration
EP2534173B1 (en) * 2010-02-08 2019-09-11 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
US20140127814A1 (en) 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
WO2012073047A2 (en) 2010-12-03 2012-06-07 Genome Research Limited Compositions and methods
ES2961613T3 (es) 2011-09-21 2024-03-12 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión transgénica
US8895264B2 (en) 2011-10-27 2014-11-25 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
RU2650811C2 (ru) * 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物

Also Published As

Publication number Publication date
CA2882499A1 (en) 2014-03-06
CA3171494A1 (en) 2014-03-06
PH12021551661A1 (en) 2022-07-18
US20180223311A1 (en) 2018-08-09
DK2890780T3 (da) 2020-09-21
BR112015003815A2 (pt) 2017-08-08
EA039384B1 (ru) 2022-01-20
KR102218562B1 (ko) 2021-02-19
LT2890780T (lt) 2020-11-10
KR20220164090A (ko) 2022-12-12
PH12019502292A1 (en) 2021-03-15
US9963715B2 (en) 2018-05-08
KR102474010B1 (ko) 2022-12-02
SI2890780T1 (sl) 2020-11-30
PH12015500433B1 (en) 2021-10-27
UA118957C2 (uk) 2019-04-10
US11492643B2 (en) 2022-11-08
AU2013308770B2 (en) 2019-01-17
HUE050516T2 (hu) 2020-12-28
EA201590167A1 (ru) 2015-11-30
KR20150047498A (ko) 2015-05-04
ES2812599T3 (es) 2021-03-17
US20140080216A1 (en) 2014-03-20
US20140093913A1 (en) 2014-04-03
CN104704110A (zh) 2015-06-10
SG11201500852WA (en) 2015-04-29
CN104704110B (zh) 2018-06-01
SG10201701601WA (en) 2017-04-27
IL237050B (en) 2020-04-30
PH12015500433A1 (en) 2015-04-20
JP2017217012A (ja) 2017-12-14
EP2890780B8 (en) 2020-08-19
WO2014036219A3 (en) 2014-05-30
HRP20201443T1 (hr) 2020-12-11
WO2014036219A2 (en) 2014-03-06
JP2018121661A (ja) 2018-08-09
JP2015533786A (ja) 2015-11-26
RS60838B1 (sr) 2020-10-30
KR20210020174A (ko) 2021-02-23
HK1210502A1 (en) 2016-04-22
MX2015002409A (es) 2015-10-05
US20230416775A1 (en) 2023-12-28
AU2013308770A1 (en) 2015-03-05
JP6629917B2 (ja) 2020-01-15
PL2890780T3 (pl) 2021-02-08
IL237050A0 (en) 2015-03-31
US9650648B2 (en) 2017-05-16
EP2890780B1 (en) 2020-06-24
PT2890780T (pt) 2020-08-03
EP2890780A2 (en) 2015-07-08
CA2882499C (en) 2023-09-26
MX367081B (es) 2019-08-05
ZA201500900B (en) 2017-09-27
JP2022001072A (ja) 2022-01-06
CL2015000450A1 (es) 2015-07-10
EP2890780A4 (en) 2016-08-31

Similar Documents

Publication Publication Date Title
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
IN2014CN02639A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
IL230836A (en) Multi-part disinfectant system
IN2012DE03088A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
LT2922554T (lt) Terminaliai modifikuota rnr
AU2013225816A8 (en) Compositions and methods for treating Type III Gaucher Disease
MX2015002487A (es) Proceso para la preparacion de teneligliptina.
SG11201407089SA (en) Cyclosporine-containing, non-irritative nanoemulsion opthalmic composition
PT2708607T (pt) Marcadores genéticos para myb28
GB201206455D0 (en) Gene expression
IN2015DN03815A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
GB2505638B (en) Continuous culture
GB201423420D0 (en) Blackhoe loader
GB201103407D0 (en) Genetic association
GB2491544A (en) Perpetum mobile
MX2012013246A (es) Composicion supresora de polvo.
GB201212331D0 (en) Elaectric pruner
UA79072U (en) (4-tert-butylperoxy-4-methylpentyl)methacrylate
AU338012S (en) Dinosaur costume
AU348528S (en) A food mould
IN2011KO00551A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
UA78206U (ru) Способ эзофагогастропластики илеоцекальным сегментом
GB201222429D0 (en) Nolan
GB201300551D0 (en) Multi-part push-pulls
IN2012DE01204A (en) "shikhar technology"