JP2017500061A5 - - Google Patents
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- JP2017500061A5 JP2017500061A5 JP2016557545A JP2016557545A JP2017500061A5 JP 2017500061 A5 JP2017500061 A5 JP 2017500061A5 JP 2016557545 A JP2016557545 A JP 2016557545A JP 2016557545 A JP2016557545 A JP 2016557545A JP 2017500061 A5 JP2017500061 A5 JP 2017500061A5
- Authority
- JP
- Japan
- Prior art keywords
- zinc finger
- composition
- expression vector
- protein
- vector
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 239000013604 expression vector Substances 0.000 claims description 17
- 102000040430 polynucleotide Human genes 0.000 claims description 12
- 108091033319 polynucleotide Proteins 0.000 claims description 12
- 239000002157 polynucleotide Substances 0.000 claims description 12
- 101710185494 Zinc finger protein Proteins 0.000 claims description 11
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims description 11
- 208000015439 Lysosomal storage disease Diseases 0.000 claims description 10
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 10
- 239000008194 pharmaceutical composition Substances 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 102000004169 proteins and genes Human genes 0.000 claims description 10
- 239000011701 zinc Substances 0.000 claims description 10
- 229910052725 zinc Inorganic materials 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 8
- 108010088751 Albumins Proteins 0.000 claims description 8
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 8
- 230000007017 scission Effects 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 108700019146 Transgenes Proteins 0.000 claims description 4
- 230000002950 deficient Effects 0.000 claims description 2
- 108020001507 fusion proteins Proteins 0.000 claims description 2
- 102000037865 fusion proteins Human genes 0.000 claims description 2
- 230000010354 integration Effects 0.000 claims description 2
- 210000005229 liver cell Anatomy 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 15
- 238000000034 method Methods 0.000 description 11
- 238000010348 incorporation Methods 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361913838P | 2013-12-09 | 2013-12-09 | |
| US61/913,838 | 2013-12-09 | ||
| US201461943884P | 2014-02-24 | 2014-02-24 | |
| US61/943,884 | 2014-02-24 | ||
| PCT/US2014/069352 WO2015089077A2 (en) | 2013-12-09 | 2014-12-09 | Methods and compositions for genome engineering |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018236131A Division JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017500061A JP2017500061A (ja) | 2017-01-05 |
| JP2017500061A5 true JP2017500061A5 (enExample) | 2017-12-21 |
| JP6535684B2 JP6535684B2 (ja) | 2019-06-26 |
Family
ID=53270543
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016557545A Expired - Fee Related JP6535684B2 (ja) | 2013-12-09 | 2014-12-09 | ゲノム操作のための方法および組成物 |
| JP2016557544A Expired - Fee Related JP6642943B2 (ja) | 2013-12-09 | 2014-12-09 | 血友病を処置するための方法および組成物 |
| JP2018236131A Pending JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016557544A Expired - Fee Related JP6642943B2 (ja) | 2013-12-09 | 2014-12-09 | 血友病を処置するための方法および組成物 |
| JP2018236131A Pending JP2019037254A (ja) | 2013-12-09 | 2018-12-18 | ゲノム操作のための方法および組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (6) | US10081661B2 (enExample) |
| EP (3) | EP3080143B1 (enExample) |
| JP (3) | JP6535684B2 (enExample) |
| CN (3) | CN105940013B (enExample) |
| AU (3) | AU2014364051B2 (enExample) |
| CA (3) | CA3229275A1 (enExample) |
| DK (1) | DK3080274T3 (enExample) |
| ES (1) | ES2813367T3 (enExample) |
| HU (1) | HUE051628T2 (enExample) |
| IL (3) | IL245785B (enExample) |
| WO (2) | WO2015089077A2 (enExample) |
Families Citing this family (101)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| ES2670983T3 (es) | 2013-11-07 | 2018-06-04 | Editas Medicine, Inc. | Métodos y composiciones relacionados con CRISPR con ARNg rectores |
| CA3229275A1 (en) | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| WO2015127439A1 (en) | 2014-02-24 | 2015-08-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| 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 |
| US10889834B2 (en) | 2014-12-15 | 2021-01-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing targeted transgene integration |
| US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| JP6954890B2 (ja) | 2015-07-13 | 2021-10-27 | サンガモ セラピューティクス， インコーポレイテッド | ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法及び組成物 |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| CN115141836B (zh) | 2015-10-28 | 2024-08-27 | 桑格摩生物治疗股份有限公司 | 肝特异性构建体、因子viii表达盒及其使用方法 |
| WO2017091512A1 (en) | 2015-11-23 | 2017-06-01 | Sangamo Biosciences, Inc. | Methods and compositions for engineering immunity |
| KR20180088911A (ko) | 2015-12-18 | 2018-08-07 | 상가모 테라퓨틱스, 인코포레이티드 | Mhc 세포 수용체의 표적화된 붕괴 |
| AU2016369490C1 (en) | 2015-12-18 | 2021-12-23 | Sangamo Therapeutics, Inc. | Targeted disruption of the T cell receptor |
| KR20180127319A (ko) | 2016-01-15 | 2018-11-28 | 상가모 테라퓨틱스, 인코포레이티드 | 신경 질환의 치료를 위한 방법 및 조성물 |
| EP3411056A4 (en) | 2016-02-02 | 2019-10-02 | Sangamo Therapeutics, Inc. | COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS |
| CN106397599B (zh) * | 2016-02-23 | 2020-08-07 | 上海交通大学 | 二价双特异性抗体杂交蛋白的表达和制备方法 |
| US11471462B2 (en) | 2016-06-27 | 2022-10-18 | The Broad Institute, Inc. | Compositions and methods for detecting and treating diabetes |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| BR112019003327A2 (pt) | 2016-08-24 | 2019-07-02 | Sangamo Therapeutics, Inc. | nucleases manipuladas de alvo específico |
| BR112019003100A2 (pt) | 2016-08-24 | 2019-07-09 | Sangamo Therapeutics, Inc. | regulação da expressão gênica usando nucleases manipuladas |
| AU2017324462B2 (en) | 2016-09-07 | 2024-03-21 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
| ES3047063T3 (en) * | 2016-10-20 | 2025-12-03 | Sangamo Therapeutics Inc | Methods and compositions for the treatment of fabry disease |
| WO2018107026A1 (en) * | 2016-12-09 | 2018-06-14 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| US11597947B2 (en) | 2016-12-29 | 2023-03-07 | Asc Therapeutics Inc. | Gene editing method using virus |
| WO2018131551A1 (ja) | 2017-01-13 | 2018-07-19 | 学校法人自治医科大学 | 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | 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 |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| CA3061612A1 (en) | 2017-04-28 | 2018-11-01 | Acuitas Therapeutics, Inc. | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| JP2020518245A (ja) | 2017-05-03 | 2020-06-25 | サンガモ セラピューティクス， インコーポレイテッド | 嚢胞性線維症膜貫通コンダクタンス制御因子（ｃｆｔｒ）遺伝子の改変のための方法および組成物 |
| 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 |
| AU2018283686A1 (en) | 2017-06-15 | 2020-01-30 | Toolgen Incorporated | Platform for expressing protein of interest in liver |
| US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
| 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 |
| BR112020002438A2 (pt) | 2017-08-08 | 2020-07-28 | Sangamo Therapeutics, Inc. | direcionamento celular mediado por receptores de antígenos quiméricos |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| BR112020007502A2 (pt) * | 2017-10-17 | 2020-10-06 | Crispr Therapeutics Ag | composições e métodos para edição gênica para hemofilia a |
| EP3704238B1 (en) * | 2017-11-01 | 2024-01-03 | Precision Biosciences, Inc. | Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019140330A1 (en) * | 2018-01-12 | 2019-07-18 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing by targeting transferrin |
| CA3089587A1 (en) * | 2018-02-08 | 2019-08-15 | Sangamo Therapeutics, Inc. | Engineered target specific nucleases |
| WO2019161310A1 (en) * | 2018-02-16 | 2019-08-22 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing by targeting fibrinogen-alpha |
| JP7522038B2 (ja) | 2018-04-06 | 2024-07-24 | ザ チルドレンズ メディカル センター コーポレーション | 体細胞リプログラミングおよびインプリンティングのモジュレートのための組成物および方法 |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| CN108795986A (zh) * | 2018-05-31 | 2018-11-13 | 深圳市免疫基因治疗研究院 | 一种a型血友病慢病毒载体、慢病毒及其制备方法和应用 |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| JP7684212B2 (ja) | 2018-08-03 | 2025-05-27 | サンガモ セラピューティクス， インコーポレイテッド | 第ｖｉｉｉ因子の発現による改善された臨床パラメーター |
| CA3106691A1 (en) * | 2018-08-07 | 2020-02-13 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type ii |
| JP7541508B2 (ja) | 2018-08-23 | 2024-08-28 | サンガモ セラピューティクス， インコーポレイテッド | 操作された標的特異的な塩基エディター |
| EP3841212A1 (en) | 2018-08-24 | 2021-06-30 | CSL Behring Gene Therapy, Inc. | Vector production in serum free media |
| KR20210060533A (ko) | 2018-09-18 | 2021-05-26 | 상가모 테라퓨틱스, 인코포레이티드 | 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제 |
| BR112021005115A2 (pt) * | 2018-09-20 | 2021-06-15 | Sanofi | métodos e composições de clonagem universal baseada em íntron |
| CA3113449A1 (en) | 2018-09-21 | 2020-03-26 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| HUE071002T2 (hu) | 2018-10-16 | 2025-07-28 | Blueallele Corp | Eljárások DNS célzott beillesztésére génekbe |
| US20210348159A1 (en) * | 2018-10-17 | 2021-11-11 | Crispr Therapeutics Ag | Compositions and methods for delivering transgenes |
| US12214023B2 (en) * | 2018-10-18 | 2025-02-04 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX |
| TWI860300B (zh) * | 2018-10-18 | 2024-11-01 | 美商英特利亞醫療公司 | 用於從白蛋白基因座表現轉殖基因的組成物及方法 |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| FI3908568T3 (fi) | 2019-01-11 | 2024-09-13 | Acuitas Therapeutics Inc | Lipidit aktiivisten aineiden lipidinanohiukkastoimitusta varten |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3826664A4 (en) * | 2019-02-06 | 2022-10-26 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| WO2020168362A1 (en) | 2019-02-15 | 2020-08-20 | Crispr Therapeutics Ag | Gene editing for hemophilia a with improved factor viii expression |
| CA3130488A1 (en) | 2019-03-19 | 2020-09-24 | David R. Liu | Methods and compositions for editing nucleotide sequences |
| CN115141807A (zh) | 2019-04-02 | 2022-10-04 | 桑格摩生物治疗股份有限公司 | 治疗β-地中海贫血症的方法 |
| CN113727603B (zh) * | 2019-04-03 | 2024-03-19 | 瑞泽恩制药公司 | 用于将抗体编码序列插入到安全港基因座中的方法和组合物 |
| US11702644B2 (en) | 2019-04-11 | 2023-07-18 | California Institute Of Technology | Methods and compositions for in vivo gene editing based cell-type-specific cellular engineering |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| JP2022533713A (ja) * | 2019-05-21 | 2022-07-25 | サンガモ セラピューティクス， インコーポレイテッド | 調節性ｔ細胞における制御された導入遺伝子発現 |
| TW202112229A (zh) | 2019-06-07 | 2021-04-01 | 美商雷傑納榮製藥公司 | 包含人化白蛋白基因座之非人的動物 |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021083073A1 (zh) * | 2019-10-31 | 2021-05-06 | 华东师范大学 | 一种基于肝细胞Alb基因的疾病治疗的产品 |
| CN114746125B (zh) | 2019-11-08 | 2025-08-08 | 瑞泽恩制药公司 | 用于x连锁青少年型视网膜劈裂症疗法的crispr和aav策略 |
| EP4146797A1 (en) | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| IL299787A (en) | 2020-07-16 | 2023-03-01 | Acuitas Therapeutics Inc | Cationic lipids for use in lipid nanoparticles |
| EP4204545A2 (en) | 2020-08-25 | 2023-07-05 | Kite Pharma, Inc. | T cells with improved functionality |
| WO2022226218A1 (en) * | 2021-04-21 | 2022-10-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Hsv amplicon packaging system using engineered cells |
| JP2024540085A (ja) * | 2021-10-27 | 2024-10-31 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 血友病ｂ療法のための第ｉｘ因子を発現させるための組成物及び方法 |
| US12129223B2 (en) | 2021-12-16 | 2024-10-29 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| US20250327120A1 (en) | 2022-01-07 | 2025-10-23 | Toolgen Incorporated | Method for predicting possible off-targets in gene editing process |
| EP4522201A1 (en) | 2022-05-09 | 2025-03-19 | 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 |
| US20250302994A1 (en) | 2022-05-09 | 2025-10-02 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified genome for gene therapy |
| WO2024197242A1 (en) | 2023-03-23 | 2024-09-26 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant vp1 capsid polypeptide and related methods |
| EP4683931A1 (en) | 2023-03-23 | 2026-01-28 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| WO2025064408A1 (en) | 2023-09-18 | 2025-03-27 | The Broad Institute, Inc. | Compositions and methods for treating cardiovascular disease |
Family Cites Families (112)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
| US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
| US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
| US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
| US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
| US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
| SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
| 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 |
| DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
| 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 |
| 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 |
| USRE39229E1 (en) | 1994-08-20 | 2006-08-08 | Gendaq Limited | Binding proteins for recognition of DNA |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| 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 |
| AU5588298A (en) | 1996-12-02 | 1998-06-29 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| 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 |
| 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 |
| EP1036177A1 (en) | 1997-12-05 | 2000-09-20 | The Immune Response Corporation | Novel vectors and genes exhibiting increased expression |
| 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 |
| EP1060261B1 (en) | 1998-03-02 | 2010-05-05 | 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 |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | 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 |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| 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 |
| JP5047437B2 (ja) | 2000-02-08 | 2012-10-10 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 薬物の発見のための細胞 |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| DK1317559T3 (da) | 2000-04-28 | 2009-03-23 | St Jude Childrens Res Hospital | DNA-transfektionssystem til frembringelse af smitsom negativstrenget RNA-virus |
| WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | 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 |
| JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Ｃｎｎについての亜鉛フィンガー結合ドメイン |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| CA2474486C (en) | 2002-01-23 | 2013-05-14 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| WO2003080809A2 (en) | 2002-03-21 | 2003-10-02 | Sangamo Biosciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US7074596B2 (en) | 2002-03-25 | 2006-07-11 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Synthesis and use of anti-reverse mRNA cap analogues |
| CA2392863A1 (en) | 2002-07-08 | 2004-01-08 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | A high capacity recombinant adenoviral vector for treatment of hemophilia a |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| AU2003298574B2 (en) | 2002-09-05 | 2008-04-24 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| AU2005233550B2 (en) | 2004-04-08 | 2010-11-18 | Sangamo Therapeutics, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| DE602005011943D1 (de) | 2004-04-08 | 2009-02-05 | Sangamo Biosciences Inc | Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen |
| US20060063231A1 (en) | 2004-09-16 | 2006-03-23 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| WO2006121866A2 (en) | 2005-05-05 | 2006-11-16 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| KR101419729B1 (ko) | 2005-07-26 | 2014-07-17 | 상가모 바이오사이언스 인코포레이티드 | 외래 핵산 서열의 표적화된 통합 및 발현 |
| ES2582091T3 (es) | 2005-10-18 | 2016-09-09 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| 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 |
| EP2447279B1 (en) | 2006-05-25 | 2014-04-09 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| WO2008109467A1 (en) | 2007-03-02 | 2008-09-12 | Arizona Board Of Regents For And On Behalf Of Arizona State University | Electrically conducting porphyrin and porphyrin-fullerene electropolymers |
| DE602008003684D1 (de) | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| PL2167523T3 (pl) | 2007-06-19 | 2014-12-31 | Univ Louisiana State | Synteza i zastosowanie anty-odwrotnych tiofosforanowych analogów kapu matrycowego RNA |
| US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
| US9506120B2 (en) | 2007-09-27 | 2016-11-29 | Sangamo Biosciences, 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 |
| EP2281050B1 (en) | 2008-04-14 | 2014-04-02 | Sangamo BioSciences, Inc. | Linear donor constructs for targeted integration |
| EP2282764B2 (en) | 2008-04-22 | 2024-06-12 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| EP2789691B1 (en) | 2008-08-22 | 2018-08-08 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| CN102625655B (zh) | 2008-12-04 | 2016-07-06 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶在大鼠中进行基因组编辑 |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| US9834787B2 (en) | 2009-04-09 | 2017-12-05 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| EP3581197A1 (de) | 2009-07-31 | 2019-12-18 | ethris GmbH | Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression |
| AU2011213242B2 (en) | 2010-02-08 | 2015-01-29 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| AU2011215557B2 (en) | 2010-02-09 | 2016-03-10 | Sangamo Therapeutics, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| SG185367A1 (en) | 2010-04-26 | 2012-12-28 | Sangamo Biosciences Inc | Genome editing of a rosa locus using zinc-finger nucleases |
| CA2798988C (en) | 2010-05-17 | 2020-03-10 | Sangamo Biosciences, Inc. | Tal-effector (tale) dna-binding polypeptides and uses thereof |
| RU2608643C2 (ru) * | 2010-10-12 | 2017-01-23 | Дзе Чилдрен`З Хоспитал Оф Филадельфия | Способы и композиции для лечения гемофилии в |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| US8601579B2 (en) * | 2011-06-03 | 2013-12-03 | Apple Inc. | System and method for preserving references in sandboxes |
| WO2013006706A1 (en) | 2011-07-05 | 2013-01-10 | Bioasis Technologies Inc. | P97-antibody conjugates and methods of use |
| AU2012312260B2 (en) | 2011-09-21 | 2017-08-31 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
| JP6188703B2 (ja) | 2011-10-27 | 2017-08-30 | サンガモ セラピューティクス， インコーポレイテッド | Ｈｐｒｔ遺伝子座を修飾するための方法および組成物 |
| EP2820127B1 (en) * | 2012-02-28 | 2018-09-26 | Sigma Aldrich Co. LLC | Targeted histone acetylation |
| JP6559063B2 (ja) * | 2012-05-07 | 2019-08-14 | サンガモ セラピューティクス， インコーポレイテッド | 導入遺伝子のヌクレアーゼ媒介標的化組み込みのための方法および組成物 |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| ES2813080T3 (es) | 2012-07-11 | 2021-03-22 | Sangamo Therapeutics Inc | Métodos y composiciones para el tratamiento de enfermedades por almacenamiento lisosomal |
| EP2911687B1 (en) | 2012-10-26 | 2019-02-13 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| US9873894B2 (en) | 2013-05-15 | 2018-01-23 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| WO2015048577A2 (en) | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| CA3229275A1 (en) * | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| US9841814B1 (en) * | 2017-01-27 | 2017-12-12 | Emergent AR Platforms Corp. | Intentional user experience |
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