HK1198178A1 - Methods and compositions for regulation of transgene expression - Google Patents

Methods and compositions for regulation of transgene expression

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Publication number
HK1198178A1
HK1198178A1 HK14111232.9A HK14111232A HK1198178A1 HK 1198178 A1 HK1198178 A1 HK 1198178A1 HK 14111232 A HK14111232 A HK 14111232A HK 1198178 A1 HK1198178 A1 HK 1198178A1
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HK
Hong Kong
Prior art keywords
regulation
compositions
methods
transgene expression
transgene
Prior art date
Application number
HK14111232.9A
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English (en)
Chinese (zh)
Inventor
．瑞巴
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Publication date
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Publication of HK1198178A1 publication Critical patent/HK1198178A1/xx

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/00—Drugs for disorders of the metabolism
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • 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
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    • C12N2510/00—Genetically modified cells
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    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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HK14111232.9A 2011-09-21 2014-11-05 Methods and compositions for regulation of transgene expression HK1198178A1 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161537349P 2011-09-21 2011-09-21
US201161560506P 2011-11-16 2011-11-16
US201261670490P 2012-07-11 2012-07-11
PCT/US2012/056539 WO2013044008A2 (en) 2011-09-21 2012-09-21 Methods and compositions for regulation of transgene expression

Publications (1)

Publication Number Publication Date
HK1198178A1 true HK1198178A1 (en) 2015-03-13

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HK14111232.9A HK1198178A1 (en) 2011-09-21 2014-11-05 Methods and compositions for regulation of transgene expression

Country Status (12)

Country Link
US (6) US9150847B2 (ko)
EP (2) EP2758529B1 (ko)
JP (3) JP6185916B2 (ko)
KR (2) KR102061557B1 (ko)
CN (2) CN103917644A (ko)
AU (3) AU2012312260B2 (ko)
CA (2) CA2848417C (ko)
ES (1) ES2961613T3 (ko)
HK (1) HK1198178A1 (ko)
IL (5) IL277027B (ko)
SG (2) SG10201811736QA (ko)
WO (1) WO2013044008A2 (ko)

Families Citing this family (143)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2848417C (en) * 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
DK3401400T3 (da) 2012-05-25 2019-06-03 Univ California Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス， インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
ES2613691T3 (es) 2012-07-11 2017-05-25 Sangamo Biosciences, Inc. Métodos y composiciones para el tratamiento de enfermedades por almacenamiento lisosomal
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014036219A2 (en) 2012-08-29 2014-03-06 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
EP3763810A3 (en) 2012-10-10 2021-07-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
RU2711249C2 (ru) * 2012-11-01 2020-01-15 Фэктор Байосайенс Инк. Способы и продукты для экспрессии белков в клетках
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
JP6620018B2 (ja) 2012-12-06 2019-12-11 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーＳｉｇｍａ−Ａｌｄｒｉｃｈ Ｃｏ．， ＬＬＣ Ｃｒｉｓｐｒに基づくゲノム修飾および制御
MX2015007550A (es) 2012-12-12 2017-02-02 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas.
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
EP2971167B1 (en) 2013-03-14 2019-07-31 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US9937207B2 (en) 2013-03-21 2018-04-10 Sangamo Therapeutics, Inc. Targeted disruption of T cell receptor genes using talens
US10604771B2 (en) * 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
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
EP4245853A3 (en) 2013-06-17 2023-10-18 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
JP6702858B2 (ja) 2013-06-17 2020-06-03 ザ・ブロード・インスティテュート・インコーポレイテッド ウイルス成分を使用して障害および疾患をターゲティングするためのＣＲＩＳＰＲ−Ｃａｓ系および組成物の送達、使用および治療上の適用
EP3011029B1 (en) 2013-06-17 2019-12-11 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
CA2915845A1 (en) * 2013-06-17 2014-12-24 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
JP6443811B2 (ja) * 2013-07-10 2018-12-26 エフストック リミテッド ライアビリティ カンパニー Ｍｒａｐ２ノックアウト
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
ES2881473T3 (es) 2013-10-17 2021-11-29 Sangamo Therapeutics Inc Métodos de suministro y composiciones para la modificación por ingeniería genética del genoma mediada por nucleasas
EP3057432B1 (en) 2013-10-17 2018-11-21 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
KR102431079B1 (ko) 2013-11-11 2022-08-11 상가모 테라퓨틱스, 인코포레이티드 헌팅턴병을 치료하기 위한 방법 및 조성물
WO2015073683A2 (en) * 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
CA2931637C (en) * 2013-12-09 2023-10-10 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
SG10201804973TA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
CN111206032A (zh) 2013-12-12 2020-05-29 布罗德研究所有限公司 用于基因组编辑的crispr-cas***和组合物的递送、用途和治疗应用
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015117081A2 (en) 2014-02-03 2015-08-06 Sangamo Biosciences, Inc. Methods and compositions for treatment of a beta thalessemia
US10370680B2 (en) * 2014-02-24 2019-08-06 Sangamo Therapeutics, Inc. Method of treating factor IX deficiency using nuclease-mediated targeted integration
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
EP3142707A4 (en) * 2014-05-13 2018-02-21 Sangamo Therapeutics, Inc. Methods and compositions for prevention or treatment of a disease
EP3151846A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9757420B2 (en) 2014-07-25 2017-09-12 Sangamo Therapeutics, Inc. Gene editing for HIV gene therapy
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
CA2960769A1 (en) 2014-09-16 2016-03-24 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
WO2016118726A2 (en) 2015-01-21 2016-07-28 Sangamo Biosciences, Inc. Methods and compositions for identification of highly specific nucleases
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MX2017014446A (es) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CN108290933A (zh) 2015-06-18 2018-07-17 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN108348576B (zh) 2015-09-23 2022-01-11 桑格摩生物治疗股份有限公司 Htt阻抑物及其用途
AU2016343803B2 (en) 2015-10-28 2021-04-29 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
AU2016343887B2 (en) * 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
US20200208173A1 (en) * 2015-10-30 2020-07-02 Seracare Life Sciences, Inc. Adenovirus control virus
CA3004349A1 (en) 2015-11-23 2017-06-01 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
WO2017100551A1 (en) * 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
EP3390437A4 (en) 2015-12-18 2019-10-16 Sangamo Therapeutics, Inc. TARGETED DISORDER OF MHC CELL RECEPTOR
JP7128741B2 (ja) 2015-12-18 2022-08-31 サンガモ セラピューティクス， インコーポレイテッド Ｔ細胞受容体の標的化破壊
CN109152847A (zh) 2016-01-15 2019-01-04 桑格摩生物治疗股份有限公司 用于治疗神经疾病的方法和组合物
EP3769775A3 (en) 2016-02-02 2021-03-17 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
US10576167B2 (en) 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
IL264639B2 (en) 2016-08-24 2024-01-01 Sangamo Therapeutics Inc Regulation of globulin gene expression using transgenic nucleases with zinc neurites
KR20220145913A (ko) 2016-08-24 2022-10-31 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
CN110022904B (zh) 2016-10-20 2024-04-19 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CA3045122A1 (en) * 2016-12-09 2018-06-14 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2018126095A1 (en) * 2016-12-29 2018-07-05 Applied Stemcell, Inc. Transgenic animals and method for bioproduction
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US11820728B2 (en) 2017-04-28 2023-11-21 Acuitas Therapeutics, Inc. Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3619322A4 (en) 2017-05-03 2021-06-30 Sangamo Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODIFICATION OF A TRANSMEMBRANARY CONDUCTANCE REGULATORY GENE IN MUCOVISCIDOSIS (CFTR)
KR20180136914A (ko) 2017-06-15 2018-12-26 주식회사 툴젠 간에서 목적하는 단백질 발현하기 위한 플랫폼
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
SG11201912235PA (en) 2017-07-31 2020-01-30 Regeneron Pharma Cas-transgenic mouse embryonic stem cells and mice and uses thereof
US20190032156A1 (en) 2017-07-31 2019-01-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
MX2020001177A (es) 2017-07-31 2020-09-25 Regeneron Pharma Animales no humanos reporteros de crispr y usos de los mismos.
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
SG11202003464VA (en) * 2017-10-17 2020-05-28 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
KR20200077529A (ko) * 2017-10-24 2020-06-30 상가모 테라퓨틱스, 인코포레이티드 희귀 질환의 치료를 위한 방법 및 조성물
WO2019094725A2 (en) 2017-11-09 2019-05-16 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible sh2-containing protein (cish) gene
CN111954540A (zh) 2018-02-08 2020-11-17 桑格摩生物治疗股份有限公司 工程化靶标特异性核酸酶
JP2021514188A (ja) 2018-02-15 2021-06-10 メモリアル スローン ケタリング キャンサー センター Ｆｏｘｐ３標的因子組成物と養子細胞療法のための使用方法
CA3089331A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
MA52656A (fr) 2018-04-05 2021-02-17 Editas Medicine Inc Procédés de production de cellules exprimant un récepteur recombinant et compositions associées
SG11202009446TA (en) 2018-04-05 2020-10-29 Juno Therapeutics Inc T cells expressing a recombinant receptor, related polynucleotides and methods
CN112313246A (zh) 2018-04-18 2021-02-02 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
BR112021001315A2 (pt) * 2018-08-03 2021-04-27 Sangamo Therapeutics, Inc. parâmetros clínicos melhorados através da expressão do fator viii
SG11202101801RA (en) 2018-08-23 2021-03-30 Sangamo Therapeutics Inc Engineered target specific base editors
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCING CD33 EXPRESSION FOR SELECTIVE PROTECTION OF THERAPEUTIC CELLS
JP2022500052A (ja) 2018-09-18 2022-01-04 サンガモ セラピューティクス， インコーポレイテッド プログラム細胞死１（ｐｄ１）特異的ヌクレアーゼ
WO2020069029A1 (en) 2018-09-26 2020-04-02 Emendobio Inc. Novel crispr nucleases
US20200270617A1 (en) 2018-10-18 2020-08-27 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
CN113272428A (zh) 2018-10-18 2021-08-17 英特利亚治疗股份有限公司 核酸构建体和使用方法
EP3886869A4 (en) 2018-11-28 2022-07-06 Forty Seven, Inc. GENETICALLY MODIFIED CSPH RESISTANT TO ABLATIVE TREATMENT
AU2020205717A1 (en) 2019-01-11 2021-08-12 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US20220098621A1 (en) 2019-02-05 2022-03-31 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
WO2020163307A1 (en) 2019-02-06 2020-08-13 Emendobio Inc. New engineered high fidelity cas9
KR20210148154A (ko) 2019-04-03 2021-12-07 리제너론 파마슈티칼스 인코포레이티드 세이프 하버 좌위 내로의 항체 코딩 서열의 삽입을 위한 방법 및 조성물
CA3136737A1 (en) 2019-05-01 2020-11-05 Juno Therapeutics, Inc. Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
BR112021021200A2 (pt) 2019-05-01 2021-12-21 Juno Therapeutics Inc Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos
AU2020289581A1 (en) 2019-06-07 2021-11-18 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
BR112021026832A2 (pt) 2019-07-02 2022-05-10 Hutchinson Fred Cancer Res Vetores ad35 recombinantes e aprimoramentos da terapia gênica relacionada
WO2021028359A1 (en) 2019-08-09 2021-02-18 Sangamo Therapeutics France Controlled expression of chimeric antigen receptors in t cells
IL292605A (en) 2019-11-08 2022-07-01 Regeneron Pharma CRISPR and AAV strategies for the treatment of x-linked childhood retinoschisis
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021195079A1 (en) 2020-03-23 2021-09-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
CN111088282B (zh) * 2020-03-23 2020-08-28 上海安民生物技术有限公司 Aavs1和h11安全港位点在重组表达蛋白中的应用
CN113061169B (zh) * 2020-03-24 2022-09-27 江南大学 一种转录调控蛋白及其在共轭亚油酸生产中的应用
WO2021231661A2 (en) 2020-05-13 2021-11-18 Juno Therapeutics, Inc. Process for producing donor-batched cells expressing a recombinant receptor
WO2021260186A1 (en) 2020-06-26 2021-12-30 Juno Therapeutics Gmbh Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods
CN116096702A (zh) 2020-07-16 2023-05-09 爱康泰生治疗公司 用于脂质纳米颗粒的阳离子脂质
EP4240756A1 (en) 2020-11-04 2023-09-13 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods
WO2022204155A1 (en) 2021-03-23 2022-09-29 Iovance Biotherapeutics, Inc. Cish gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
KR20220136677A (ko) * 2021-04-01 2022-10-11 서울대학교산학협력단 인간 면역 글로불린 Fc 및 목적단백질을 생산하는 조류의 제조방법
CN115485367B (zh) * 2021-04-16 2023-09-12 杭州启函生物科技有限公司 用于细胞工程的安全港基因座
CA3232470A1 (en) 2021-10-27 2023-05-04 Leah SABIN Compositions and methods for expressing factor ix for hemophilia b therapy
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
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
WO2023108047A1 (en) 2021-12-08 2023-06-15 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
WO2023129940A1 (en) 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023150620A1 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Crispr-mediated transgene insertion in neonatal cells
WO2023150393A2 (en) 2022-02-07 2023-08-10 Ensoma, Inc. Inhibitor-resistant mgmt modifications and modification of mgmt-encoding nucleic acids
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
WO2023154861A1 (en) 2022-02-11 2023-08-17 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
WO2023220035A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024073606A1 (en) 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024100604A1 (en) 2022-11-09 2024-05-16 Juno Therapeutics Gmbh Methods for manufacturing engineered immune cells
US20240173426A1 (en) 2022-11-14 2024-05-30 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes

Family Cites Families (104)

* 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
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (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
CA2681922C (en) 1994-01-18 2012-05-15 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
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
ES2316148T3 (es) 1994-03-23 2009-04-01 Ohio University Acidos nucleicos compactados y su suministro a celulas.
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
JP4118327B2 (ja) 1994-08-20 2008-07-16 ゲンダック・リミテッド Ｄｎａ認識のための結合タンパク質におけるまたはそれに関連する改良
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
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
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
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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
US6824987B1 (en) 1999-05-11 2004-11-30 President And Fellows Of Harvard College Small molecule printing
US6689558B2 (en) 2000-02-08 2004-02-10 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
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
CA2416664A1 (en) 2000-07-21 2002-01-31 Syngenta Participations Ag Zinc finger domain recognition code and uses thereof
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
AUPR446701A0 (en) 2001-04-18 2001-05-17 Gene Stream Pty Ltd Transgenic mammals for pharmacological and toxicological studies
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
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
BRPI0307383B1 (pt) 2002-01-23 2019-12-31 The Univ Of Utah Research Foundation método de recombinação genética direcionada em célula de planta hospedeira
WO2003080809A2 (en) 2002-03-21 2003-10-02 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
WO2004037977A2 (en) 2002-09-05 2004-05-06 California Institute Of Thechnology Use of chimeric nucleases to stimulate gene 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
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1737498A2 (en) * 2004-04-08 2007-01-03 Sangamo Biosciences Inc. Zinc finger proteins for treatment of neuropathic pain
CA2561714A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US20080131962A1 (en) 2006-05-25 2008-06-05 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2006033859A2 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
AU2005295033B2 (en) * 2004-10-08 2012-01-19 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
US20090068164A1 (en) 2005-05-05 2009-03-12 The Ariz Bd Of Regents On Behald Of The Univ Of Az Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
US8043088B2 (en) 2005-05-16 2011-10-25 Johnson Douglas B Endodontic procedure employing simultaneous liquefaction and acoustic debridgement
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
JP5937292B2 (ja) 2005-10-18 2016-06-22 デューク大学 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ
AU2007267874B2 (en) 2006-05-25 2012-03-15 Sangamo Therapeutics, Inc. Methods and compositions for gene inactivation
CA2660485C (en) 2006-08-11 2016-04-12 Sangamo Biosciences, Inc. Zinc finger nuclease-mediated homologous recombination
JP2010516277A (ja) * 2007-01-26 2010-05-20 シナジェバ・バイオファーマ・コーポレイション トリにおける導入遺伝子発現
WO2008091311A1 (en) 2007-01-26 2008-07-31 Synageva Biopharma Corp Transgene expression in avians
ATE489465T1 (de) 2007-04-26 2010-12-15 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
NZ581396A (en) * 2007-06-01 2012-07-27 Omt Inc Compositions and methods for inhibiting endogenous immunoglobulin genes and producing transgenic human idiotype antibodies
EP2006388A1 (de) * 2007-06-19 2008-12-24 Helmholtz-Zentrum für Infektionsforschung GmbH Nukleinsäurekonstrukte zur Inaktivierung und konditionalen Mutagenese von Genen
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
WO2009042163A2 (en) 2007-09-27 2009-04-02 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
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
AU2009260888B2 (en) 2008-05-28 2014-09-11 Sangamo Therapeutics, 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
US20110023158A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Bovine genome editing with zinc finger nucleases
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
WO2011011767A1 (en) * 2009-07-24 2011-01-27 Sigma-Aldrich Co. Method for genome editing
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
KR102262704B1 (ko) 2009-08-11 2021-06-09 상가모 테라퓨틱스, 인코포레이티드 표적화 변형에 대한 동형접합성 유기체
FR2953287B1 (fr) 2009-11-27 2017-07-21 Mc Aci Dispositif de mesure de grandeurs parietales
CN102770539B (zh) 2009-12-10 2016-08-03 明尼苏达大学董事会 Tal效应子介导的dna修饰
US8962281B2 (en) 2010-02-08 2015-02-24 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP6018069B2 (ja) 2010-10-12 2016-11-02 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 血友病ｂを治療する方法及び組成物
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
JP2016511126A (ja) 2013-03-15 2016-04-14 マイクロベンション インコーポレイテッド 多要素障害物除去システムおよび方法

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US20130177983A1 (en) 2013-07-11
IL263324A (en) 2018-12-31
IL293650A (en) 2022-08-01
IL262238B (en) 2021-03-25
IL277027B (en) 2022-07-01
EP2758529B1 (en) 2019-03-20
US9777281B2 (en) 2017-10-03
KR20190099343A (ko) 2019-08-26
AU2012312260A1 (en) 2014-03-27
JP2017217002A (ja) 2017-12-14
KR102209330B1 (ko) 2021-01-29
IL277027A (en) 2020-10-29
SG11201400707TA (en) 2014-04-28
CN103917644A (zh) 2014-07-09
US9394545B2 (en) 2016-07-19
AU2019206021A1 (en) 2019-08-01
IL293650B1 (en) 2024-06-01
JP6496363B2 (ja) 2019-04-03
ES2961613T3 (es) 2024-03-12
CA2848417C (en) 2023-05-02
US20150344893A1 (en) 2015-12-03
EP3498833B1 (en) 2023-08-16
WO2013044008A2 (en) 2013-03-28
KR102061557B1 (ko) 2020-01-03
CA2848417A1 (en) 2013-03-28
US20170355999A1 (en) 2017-12-14
EP3498833A1 (en) 2019-06-19
KR20140069205A (ko) 2014-06-09
NZ622180A (en) 2016-03-31
AU2017264986B2 (en) 2019-05-16
JP2014526279A (ja) 2014-10-06
US10975375B2 (en) 2021-04-13
NZ718174A (en) 2017-03-31
SG10201811736QA (en) 2019-02-27
US11639504B2 (en) 2023-05-02
AU2019206021B2 (en) 2021-07-15
JP6890145B2 (ja) 2021-06-18
CA3186126A1 (en) 2013-03-28
US11859190B2 (en) 2024-01-02
NZ707974A (en) 2016-06-24
US20130177960A1 (en) 2013-07-11
EP3498833C0 (en) 2023-08-16
US20190218560A1 (en) 2019-07-18
US20210189405A1 (en) 2021-06-24
AU2012312260B2 (en) 2017-08-31
EP2758529A2 (en) 2014-07-30
US9150847B2 (en) 2015-10-06
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AU2017264986A1 (en) 2017-12-07
JP2019080588A (ja) 2019-05-30

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