JP2015533786A - 遺伝子疾患の治療のための方法および組成物 - Google Patents

遺伝子疾患の治療のための方法および組成物 Download PDF

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JP2015533786A
JP2015533786A JP2015530017A JP2015530017A JP2015533786A JP 2015533786 A JP2015533786 A JP 2015533786A JP 2015530017 A JP2015530017 A JP 2015530017A JP 2015530017 A JP2015530017 A JP 2015530017A JP 2015533786 A JP2015533786 A JP 2015533786A
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globin
gene
cell
sequence
cells
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JP2015533786A5 (enExample
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グレゴリー ジェイ. コスト,
グレゴリー ジェイ. コスト,
フィリップ ディー. グレゴリー,
フィリップ ディー. グレゴリー,
ドミトリー ガスチン,
ドミトリー ガスチン,
マイケル シー. ホームズ,
マイケル シー. ホームズ,
ジェフリー シー. ミラー,
ジェフリー シー. ミラー,
デイビッド パッション,
デイビッド パッション,
エドワード ジェイ. リーバー,
エドワード ジェイ. リーバー,
アンドレアス ライク,
アンドレアス ライク,
フィオードル ウルノフ,
フィオードル ウルノフ,
レイ ザン,
レイ ザン,
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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JP2015530017A 2012-08-29 2013-08-29 遺伝子疾患の治療のための方法および組成物 Pending JP2015533786A (ja)

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US201261694693P 2012-08-29 2012-08-29
US61/694,693 2012-08-29
PCT/US2013/057214 WO2014036219A2 (en) 2012-08-29 2013-08-29 Methods and compositions for treatment of a genetic condition

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JP2017180131A Pending JP2017217012A (ja) 2012-08-29 2017-09-20 遺伝子疾患の治療のための方法および組成物
JP2018093930A Expired - Fee Related JP6629917B2 (ja) 2012-08-29 2018-05-15 遺伝子疾患の治療のための方法および組成物
JP2021169438A Pending JP2022001072A (ja) 2012-08-29 2021-10-15 遺伝子疾患の治療のための方法および組成物

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JP2021169438A Pending JP2022001072A (ja) 2012-08-29 2021-10-15 遺伝子疾患の治療のための方法および組成物

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022528744A (ja) * 2019-04-12 2022-06-15 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作された造血幹細胞によるベータサラセミア表現型の補正
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

Families Citing this family (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
EP3272356A1 (en) 2012-02-24 2018-01-24 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CN110669758A (zh) 2012-10-23 2020-01-10 基因工具股份有限公司 用于切割靶dna 的组合物及其用途
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
DK3620534T3 (da) 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝的状態の処置のための方法および組成物
CN105658805B (zh) * 2013-06-05 2021-12-31 杜克大学 Rna指导的基因编辑和基因调节
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
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
JP6588438B2 (ja) 2013-08-28 2019-10-09 サンガモ セラピューティクス, インコーポレイテッド Dna結合ドメインと切断ドメインとを連結するための組成物
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
EP3066201B1 (en) 2013-11-07 2018-03-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
PT3068881T (pt) * 2013-11-13 2019-05-31 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
US10072066B2 (en) * 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
JP6606088B2 (ja) 2014-02-24 2019-11-13 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介性標的化組み込みのための方法および組成物
WO2015134812A1 (en) 2014-03-05 2015-09-11 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
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
CA2942762C (en) 2014-03-18 2023-10-17 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
US20170173083A1 (en) * 2014-03-26 2017-06-22 The Brigham And Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
EP3981876A1 (en) * 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
WO2016022363A2 (en) 2014-07-30 2016-02-11 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
US10435677B2 (en) * 2014-09-16 2019-10-08 Sangamo Therapeutics, Inc. Genetically modified human cell with a corrected mutant sickle cell mutation
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
US10395760B2 (en) 2015-04-13 2019-08-27 Invitae Corporation Methods, systems and processes of identifying genetic variation in highly similar genes
US11180793B2 (en) 2015-04-24 2021-11-23 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
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
US10808020B2 (en) * 2015-05-12 2020-10-20 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
WO2017015637A1 (en) * 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
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
AU2016341041A1 (en) * 2015-10-20 2018-03-15 Pioneer Hi-Bred International, Inc. Methods and compositions for marker-free genome modification
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
KR20250022906A (ko) 2015-10-28 2025-02-17 상가모 테라퓨틱스, 인코포레이티드 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
EP3374502B1 (en) 2015-11-13 2021-10-27 Avellino Lab USA, Inc. Methods for the treatment of corneal dystrophies
WO2017106537A2 (en) 2015-12-18 2017-06-22 Sangamo Biosciences, Inc. Targeted disruption of the mhc cell receptor
EP3417061B1 (en) 2016-02-18 2022-10-26 The Regents of the University of California Methods and compositions for gene editing in stem cells
CN118127006A (zh) * 2016-03-14 2024-06-04 爱迪塔斯医药公司 用于治疗β-血红蛋白病的CRISPR/CAS相关方法和组合物
WO2017165862A1 (en) 2016-03-25 2017-09-28 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
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard 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
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
ES2901989T3 (es) 2016-08-24 2022-03-24 Sangamo Therapeutics Inc Nucleasas específicas de objetivo modificadas
MX2019002207A (es) 2016-08-24 2019-07-15 Sangamo Therapeutics Inc Regulacion de la expresion genica usando nucleasas modificadas.
AU2017324462B2 (en) 2016-09-07 2024-03-21 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
US20190330620A1 (en) * 2016-10-14 2019-10-31 Emendobio Inc. Rna compositions for genome editing
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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
EP3655534B1 (en) 2017-07-18 2025-07-16 CSL Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies
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
US11319532B2 (en) 2017-08-30 2022-05-03 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
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
IL274323B2 (en) * 2017-11-09 2024-10-01 Sangamo Therapeutics Inc Genetic modification of cytokine inducible sh2-containing protein (cish) gene
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3511412A1 (en) * 2018-01-12 2019-07-17 Genethon Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
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
AU2019225992A1 (en) 2018-02-21 2020-08-20 Alcyone Therapeutics, Inc. Fluid delivery systems and methods
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN112534054B (zh) 2018-05-11 2025-10-28 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
WO2020014261A1 (en) 2018-07-09 2020-01-16 The Broad Institute, Inc. Rna programmable epigenetic rna modifiers and uses thereof
WO2020046791A1 (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
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
BR112021018607A2 (pt) 2019-03-19 2021-11-23 Massachusetts Inst Technology Métodos e composições para editar sequências de nucleotídeos
WO2020205838A1 (en) 2019-04-02 2020-10-08 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
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
US20220280571A1 (en) * 2019-08-01 2022-09-08 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
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021076592A1 (en) * 2019-10-15 2021-04-22 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
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2023115039A2 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
EP4448775A1 (en) 2021-12-17 2024-10-23 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
US20250152709A1 (en) 2022-02-01 2025-05-15 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
EP4473097A1 (en) 2022-02-02 2024-12-11 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
WO2024064838A1 (en) 2022-09-21 2024-03-28 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
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
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
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
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002534104A (ja) * 1999-01-12 2002-10-15 サンガモ バイオサイエンシーズ, インコーポレイテッド ジンクフィンガータンパク質を用いた、細胞における、内因性遺伝子発現の調節
JP2007501626A (ja) * 2003-08-08 2007-02-01 サンガモ バイオサイエンシズ インコーポレイテッド 標的化された切断及び組換えの方法及び組成物
JP2008506359A (ja) * 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
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
WO2011097036A1 (en) * 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains

Family Cites Families (100)

* 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
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
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
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
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii 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
US5877302A (en) 1994-03-23 1999-03-02 Case Western Reserve University Compacted nucleic acids and their delivery to cells
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
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
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
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
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
EP1147209A2 (en) 1999-02-03 2001-10-24 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
WO2001040798A2 (en) 1999-12-06 2001-06-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
WO2001059450A2 (en) 2000-02-08 2001-08-16 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
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
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
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F 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
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
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
CA2497913C (en) 2002-09-05 2014-06-03 California Institute Of Technology 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
JP4903689B2 (ja) 2004-04-08 2012-03-28 サンガモ バイオサイエンシズ インコーポレイテッド 神経障害および神経変性症状を治療するための方法と組成物
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
EP2314614B1 (en) * 2005-02-28 2015-11-25 Sangamo BioSciences, Inc. Anti-angiogenic methods and compositions
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
EP2628794B1 (en) 2005-10-18 2016-05-04 Precision Biosciences Rationally designed meganucleases with altered sequence specificity and DNA-binding affinity
WO2007139982A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Methods and compositions for gene inactivation
AU2007267887B2 (en) 2006-05-25 2013-05-30 Sangamo Therapeutics, Inc. Variant Foki Cleavage Half-Domains
ATE489465T1 (de) 2007-04-26 2010-12-15 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
KR101657504B1 (ko) 2007-09-27 2016-09-19 상가모 바이오사이언스 인코포레이티드 생물학적으로 활성인 뉴클레아제의 신속한 생체내 확인
US8936936B2 (en) 2007-10-25 2015-01-20 Sangamo Biosciences, Inc. 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
CA2720903C (en) 2008-04-14 2019-01-15 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
CA2725773C (en) 2008-05-28 2017-12-05 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
ES2615829T3 (es) 2008-09-15 2017-06-08 The Children's Medical Center Corporation Modulación de BCL11A para el tratamiento de hemoglobinopatías
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
EP3156504B1 (en) 2009-08-11 2018-10-31 Sangamo Therapeutics, Inc. Organisms homozygous for targeted modification
DK2510096T4 (en) 2009-12-10 2018-05-14 Univ Iowa State Res Found Inc NUMBER EFFECTOR-MEDIATED DNA MODIFICATION
MX336846B (es) 2010-01-22 2016-02-03 Sangamo Biosciences Inc Alteracion genomica dirigida.
AU2011215557B2 (en) 2010-02-09 2016-03-10 Sangamo Therapeutics, Inc. Targeted genomic modification with partially single-stranded donor molecules
SI2566972T1 (sl) 2010-05-03 2020-04-30 Sangamo Therapeutics, Inc. Sestavki za povezovanje modulov cinkovega prsta
KR101953237B1 (ko) 2010-05-17 2019-02-28 상가모 테라퓨틱스, 인코포레이티드 신규 dna 결합 단백질 및 이의 용도
WO2012021632A2 (en) 2010-08-10 2012-02-16 The Johns Hopkins University 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
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
EP3272356A1 (en) * 2012-02-24 2018-01-24 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝的状態の処置のための方法および組成物

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002534104A (ja) * 1999-01-12 2002-10-15 サンガモ バイオサイエンシーズ, インコーポレイテッド ジンクフィンガータンパク質を用いた、細胞における、内因性遺伝子発現の調節
JP2007501626A (ja) * 2003-08-08 2007-02-01 サンガモ バイオサイエンシズ インコーポレイテッド 標的化された切断及び組換えの方法及び組成物
JP2008506359A (ja) * 2004-04-08 2008-03-06 サンガモ バイオサイエンシズ インコーポレイテッド ジンクフィンガータンパク質による神経因性疼痛の処置
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
WO2011097036A1 (en) * 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BMC BLOOD DISORDERS, 2012, 12:5, P.1-8, JPN6017023079, ISSN: 0003742670 *
STEM CELLS, 2011, 29:1717-1726, JPN6017023080, ISSN: 0003583159 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022528744A (ja) * 2019-04-12 2022-06-15 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作された造血幹細胞によるベータサラセミア表現型の補正
JP7469328B2 (ja) 2019-04-12 2024-04-16 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 遺伝子操作された造血幹細胞によるベータサラセミア表現型の補正
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

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