JP2017500061A5 - - Google Patents
Download PDFInfo
- Publication number
- 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.)
- Granted
Links
- 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 |
|---|---|---|---|
| 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 |
| 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 |
| 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 Before (1)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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) | US9771403B2 (enExample) |
| EP (3) | EP3080274B1 (enExample) |
| JP (3) | JP6642943B2 (enExample) |
| CN (3) | CN105874071B (enExample) |
| AU (3) | AU2014364051B2 (enExample) |
| CA (3) | CA3229275A1 (enExample) |
| DK (1) | DK3080274T3 (enExample) |
| ES (1) | ES2813367T3 (enExample) |
| HU (1) | HUE051628T2 (enExample) |
| IL (3) | IL245778B (enExample) |
| WO (2) | WO2015089046A1 (enExample) |
Families Citing this family (102)
| 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 |
| 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| JP2016536021A (ja) | 2013-11-07 | 2016-11-24 | エディタス・メディシン，インコーポレイテッド | ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物 |
| EP3080274B1 (en) | 2013-12-09 | 2020-06-03 | Sangamo Therapeutics, Inc. | Methods and compositions for genome engineering |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| AU2015218576B2 (en) * | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| CA2956224A1 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| AU2015308910B2 (en) | 2014-08-27 | 2017-12-07 | 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 |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| EP4434589A3 (en) | 2015-10-23 | 2025-05-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| KR102766526B1 (ko) | 2015-10-28 | 2025-02-13 | 상가모 테라퓨틱스, 인코포레이티드 | 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 |
| JP6976249B2 (ja) | 2015-11-23 | 2021-12-08 | サンガモ セラピューティクス， インコーポレイテッド | 免疫を工学操作するための方法および組成物 |
| WO2017106528A2 (en) | 2015-12-18 | 2017-06-22 | Sangamo Biosciences, Inc. | Targeted disruption of the t cell receptor |
| MX2018007519A (es) | 2015-12-18 | 2019-09-04 | Sangamo Therapeutics Inc | Alteracion dirigida del receptor celular del complejo mayor de histocompatibilidad (mhc). |
| ES2895430T3 (es) * | 2016-01-15 | 2022-02-21 | Univ Minnesota | Métodos y composiciones para el tratamiento de enfermedad neurológica |
| JP7012650B2 (ja) | 2016-02-02 | 2022-01-28 | 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 |
| 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 |
| EP3474849B1 (en) | 2016-06-27 | 2025-05-21 | The Broad Institute, Inc. | Compositions and methods for detecting and treating diabetes |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | 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 | アデノシン核酸塩基編集因子およびそれらの使用 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| CR20190098A (es) * | 2016-08-24 | 2019-04-30 | Sangamo Therapeutics Inc | Regulación de la expresión génica usando nucleasas modificadas |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| SG11201901364VA (en) | 2016-08-24 | 2019-03-28 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
| WO2018049009A2 (en) * | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| JP7042263B2 (ja) * | 2016-10-20 | 2022-03-25 | サンガモ セラピューティクス， インコーポレイテッド | ファブリー病の治療のための方法および組成物 |
| AU2017374042C1 (en) * | 2016-12-09 | 2024-07-11 | Acuitas Therapeutics, Inc. | Delivery of target specific nucleases |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3562515A4 (en) * | 2016-12-29 | 2020-11-18 | Applied StemCell, Inc. | GENE EDITING PROCESS USING A VIRUS |
| JP7215716B2 (ja) * | 2017-01-13 | 2023-01-31 | 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 |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| 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 |
| WO2018176009A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018200943A1 (en) | 2017-04-28 | 2018-11-01 | Acuitas Therapeutics, Inc. | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| CA3059793A1 (en) | 2017-05-03 | 2018-11-08 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
| 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 |
| KR102738651B1 (ko) | 2017-06-15 | 2024-12-05 | 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 |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| WO2019032675A1 (en) | 2017-08-08 | 2019-02-14 | Sangamo Therapeutics, Inc. | CELL TARGETING MEDIATED BY A CHIMERIC ANTIGEN RECEPTOR |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| KR20200121782A (ko) | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| CN111684070A (zh) * | 2017-10-17 | 2020-09-18 | 克里斯珀医疗股份公司 | 用于a型血友病基因编辑的组合物和方法 |
| WO2019089913A1 (en) * | 2017-11-01 | 2019-05-09 | 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 |
| EP3737762A1 (en) | 2018-01-12 | 2020-11-18 | CRISPR Therapeutics AG | Compositions and methods for gene editing by targeting transferrin |
| MX2020008272A (es) * | 2018-02-08 | 2020-09-21 | Sangamo Therapeutics Inc | Nucleasas diseñadas específicas de blancos. |
| WO2019161310A1 (en) * | 2018-02-16 | 2019-08-22 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing by targeting fibrinogen-alpha |
| US20210155959A1 (en) | 2018-04-06 | 2021-05-27 | Children's Medical Center Corporation | Compositions and methods for somatic cell reprogramming and modulating imprinting |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | 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 |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| 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 |
| EP3833752A4 (en) * | 2018-08-07 | 2022-06-01 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type ii |
| KR102712986B1 (ko) | 2018-08-23 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 조작된 표적 특이적 염기 편집기 |
| WO2020041647A1 (en) | 2018-08-24 | 2020-02-27 | Calimmune, Inc. | Vector production in serum free media |
| AU2019344927A1 (en) | 2018-09-18 | 2021-04-01 | Sangamo Therapeutics, Inc. | Programmed cell death 1 (PD1) specific nucleases |
| MX2021003273A (es) * | 2018-09-20 | 2021-07-16 | Sanofi Sa | Metodos y composiciones de clonacion universal basados en intrones. |
| EP4613367A1 (en) | 2018-09-21 | 2025-09-10 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| JP7520826B2 (ja) * | 2018-10-17 | 2024-07-23 | クリスパー・セラピューティクス・アクチェンゲゼルシャフト | 導入遺伝子を送達するための組成物および方法 |
| EP3867380A2 (en) * | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| MX2021004278A (es) * | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para la expresion de transgenes a partir de un locus de albumina. |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CA3125485A1 (en) | 2019-01-11 | 2020-07-16 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | 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 |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| WO2020205838A1 (en) | 2019-04-02 | 2020-10-08 | Sangamo Therapeutics, Inc. | Methods for the treatment of beta-thalassemia |
| AU2020256225B9 (en) * | 2019-04-03 | 2025-04-10 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for insertion of antibody coding sequences into a safe harbor locus |
| WO2020210552A1 (en) * | 2019-04-11 | 2020-10-15 | California Institute Of Technology | Methods and compositions for in vivo gene editing based cell-type-specific cellular engineering |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3141159A1 (en) * | 2019-05-21 | 2020-11-26 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| AU2020289581B2 (en) | 2019-06-07 | 2024-11-21 | Regeneron Pharmaceuticals, Inc. | Non-human animals comprising a humanized albumin locus |
| CN114245803A (zh) | 2019-08-06 | 2022-03-25 | 儿童医疗中心有限公司 | 用于重构小神经胶质细胞的方法和组合物 |
| 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 |
| AU2020379046B2 (en) | 2019-11-08 | 2025-03-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for X-linked juvenile retinoschisis therapy |
| WO2021224633A1 (en) | 2020-05-06 | 2021-11-11 | 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 |
| DK4182297T3 (da) | 2020-07-16 | 2025-12-01 | Acuitas Therapeutics Inc | Kationiske lipider til brug i lipid-nanopartikler |
| US12152251B2 (en) | 2020-08-25 | 2024-11-26 | Kite Pharma, Inc. | T cells with improved functionality |
| US20240191243A1 (en) * | 2021-04-21 | 2024-06-13 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Hsv amplicon packaging system using engineered cells |
| WO2023077012A1 (en) * | 2021-10-27 | 2023-05-04 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
| WO2023114943A2 (en) | 2021-12-16 | 2023-06-22 | Acuitas Therapeutics, Inc. | Lipids for use in lipid nanoparticle formulations |
| WO2023132704A1 (ko) | 2022-01-07 | 2023-07-13 | 주식회사 툴젠 | 유전자 편집 과정에서 발생 가능한 오프 타겟을 예측하는 방법 |
| WO2023220043A1 (en) | 2022-05-09 | 2023-11-16 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified genome for gene therapy |
| 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 |
| 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 |
| 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 |
| 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 |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| CA2181548C (en) | 1994-01-18 | 2009-11-03 | Carlos F. Barbas, Iii | Zinc finger protein derivatives and methods therefor |
| JP4285766B2 (ja) | 1994-03-23 | 2009-06-24 | オハイオ ユニバーシティ | 緻密にした核酸および細胞へのデリバリ |
| 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 |
| DE69535829D1 (de) | 1994-08-20 | 2008-10-16 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von 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 |
| 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 |
| 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 |
| JP2002517180A (ja) | 1997-12-05 | 2002-06-18 | ジ・イミユーン・リスポンス・コーポレーシヨン | 増大された発現を示す新規ベクターおよび遺伝子 |
| 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 |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
| 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 |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| 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 |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| CA2394850C (en) * | 1999-12-06 | 2012-02-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| EP1254369B1 (en) | 2000-02-08 | 2010-10-06 | 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 |
| CN100489107C (zh) | 2000-04-28 | 2009-05-20 | 圣朱德儿童研究医院 | 用于产生感染性流感病毒的dna转染系统 |
| 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 |
| WO2003016496A2 (en) | 2001-08-20 | 2003-02-27 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| 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 |
| US7238346B2 (en) | 2002-07-08 | 2007-07-03 | Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw | 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 |
| WO2004037977A2 (en) | 2002-09-05 | 2004-05-06 | California Institute Of Thechnology | 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 |
| US7253273B2 (en) | 2004-04-08 | 2007-08-07 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| 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 |
| 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 |
| EP1913149A4 (en) | 2005-07-26 | 2009-08-05 | Sangamo Biosciences Inc | TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES |
| US20090263900A1 (en) | 2008-04-14 | 2009-10-22 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| CA2626262C (en) | 2005-10-18 | 2015-09-08 | Homme W. Hellinga | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| WO2007120533A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| DE602007005634D1 (de) | 2006-05-25 | 2010-05-12 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
| EP2019839B1 (en) | 2006-05-25 | 2011-12-07 | 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 |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| DK2167523T3 (da) | 2007-06-19 | 2014-09-08 | Univ Louisiana State | Syntese og anvendelse af anti-reverse phosphorothioatanaloger af messenger-rna-cappen |
| US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
| CN101883863B (zh) | 2007-09-27 | 2018-01-23 | 桑格摩生物科学股份有限公司 | 生物活性核酸酶的快速体内鉴定 |
| CA2703045C (en) | 2007-10-25 | 2017-02-14 | Sangamo Biosciences, Inc. | Methods and compositions for targeted integration |
| DK2282764T3 (da) | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| SG191561A1 (en) | 2008-08-22 | 2013-07-31 | Sangamo Biosciences Inc | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| JP5681114B2 (ja) | 2008-12-04 | 2015-03-04 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集 |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| JP6215533B2 (ja) | 2009-04-09 | 2017-10-18 | サンガモ セラピューティクス， インコーポレイテッド | 幹細胞への標的組込み |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| BR112012002291A2 (pt) | 2009-07-31 | 2016-11-29 | Ethris Gmbh | "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos" |
| DK2534173T3 (da) | 2010-02-08 | 2019-10-14 | Sangamo Therapeutics Inc | Manipulerede spaltnings-halvdomæner |
| EP2534163B1 (en) | 2010-02-09 | 2015-11-04 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| KR101880536B1 (ko) | 2010-04-26 | 2018-07-23 | 상가모 테라퓨틱스, 인코포레이티드 | 아연 핑거 뉴클레아제를 사용하는 로사 좌위의 게놈 편집 |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
| CN103270046B (zh) | 2010-10-12 | 2016-05-11 | 费城儿童医院 | 治疗b型血友病的方法和组合物 |
| 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 |
| AU2012278944B2 (en) | 2011-07-05 | 2015-09-17 | Bioasis Technologies Inc. | p97-antibody conjugates and methods of use |
| CA2848417C (en) * | 2011-09-21 | 2023-05-02 | 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 |
| JP6309461B2 (ja) * | 2012-02-28 | 2018-04-11 | 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 | ヒストンアセチル化の標的化 |
| CA2871524C (en) * | 2012-05-07 | 2021-07-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration of transgenes |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| DK2911687T3 (da) | 2012-10-26 | 2019-05-13 | Univ Brussel Vrije | Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf |
| 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 |
| WO2015048577A2 (en) | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| EP3080274B1 (en) * | 2013-12-09 | 2020-06-03 | Sangamo Therapeutics, Inc. | Methods and compositions for genome engineering |
| US9841814B1 (en) * | 2017-01-27 | 2017-12-12 | Emergent AR Platforms Corp. | Intentional user experience |
-
2014
- 2014-12-09 EP EP14869719.6A patent/EP3080274B1/en active Active
- 2014-12-09 EP EP20177689.5A patent/EP3757116A1/en not_active Withdrawn
- 2014-12-09 CA CA3229275A patent/CA3229275A1/en active Pending
- 2014-12-09 US US14/564,722 patent/US9771403B2/en not_active Expired - Fee Related
- 2014-12-09 ES ES14869719T patent/ES2813367T3/es active Active
- 2014-12-09 CN CN201480067248.8A patent/CN105874071B/zh not_active Expired - Fee Related
- 2014-12-09 JP JP2016557544A patent/JP6642943B2/ja not_active Expired - Fee Related
- 2014-12-09 CN CN202010184376.8A patent/CN111330021A/zh active Pending
- 2014-12-09 JP JP2016557545A patent/JP6535684B2/ja not_active Expired - Fee Related
- 2014-12-09 DK DK14869719.6T patent/DK3080274T3/da active
- 2014-12-09 CA CA2931637A patent/CA2931637C/en active Active
- 2014-12-09 US US14/565,014 patent/US10081661B2/en active Active
- 2014-12-09 HU HUE14869719A patent/HUE051628T2/hu unknown
- 2014-12-09 EP EP14869305.4A patent/EP3080143B1/en active Active
- 2014-12-09 CN CN201480072482.XA patent/CN105940013B/zh not_active Expired - Fee Related
- 2014-12-09 WO PCT/US2014/069307 patent/WO2015089046A1/en not_active Ceased
- 2014-12-09 CA CA2931848A patent/CA2931848A1/en active Pending
- 2014-12-09 WO PCT/US2014/069352 patent/WO2015089077A2/en not_active Ceased
- 2014-12-09 AU AU2014364051A patent/AU2014364051B2/en not_active Ceased
- 2014-12-09 AU AU2014363991A patent/AU2014363991B2/en not_active Ceased
-
2016
- 2016-05-23 IL IL245778A patent/IL245778B/en active IP Right Grant
- 2016-05-23 IL IL245785A patent/IL245785B/en active IP Right Grant
-
2017
- 2017-08-09 US US15/673,163 patent/US10407476B2/en active Active
-
2018
- 2018-08-21 US US16/107,891 patent/US10968261B2/en not_active Expired - Fee Related
- 2018-12-18 JP JP2018236131A patent/JP2019037254A/ja active Pending
-
2019
- 2019-07-26 US US16/523,328 patent/US11634463B2/en active Active
-
2020
- 2020-09-07 IL IL277196A patent/IL277196B/en unknown
- 2020-11-23 AU AU2020277091A patent/AU2020277091C1/en not_active Ceased
-
2021
- 2021-03-01 US US17/188,764 patent/US20210188926A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2017500061A5 (enExample) | ||
| JP2017504354A5 (enExample) | ||
| Erkut et al. | CRISPR therapeutics for Duchenne muscular dystrophy | |
| Tipanee et al. | Transposons: moving forward from preclinical studies to clinical trials | |
| Budamagunta et al. | Cellular senescence in lymphoid organs and immunosenescence | |
| Hamanaka et al. | Generation of vascular endothelial cells and hematopoietic cells by blastocyst complementation | |
| JP2016539627A5 (enExample) | ||
| Wang et al. | Knocking out non-muscle myosin II in retinal ganglion cells promotes long-distance optic nerve regeneration | |
| Huai et al. | CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice | |
| JP2013544077A5 (enExample) | ||
| Ebert et al. | Reprogramming and transdifferentiation for cardiovascular development and regenerative medicine: where do we stand? | |
| JP2019524150A5 (enExample) | ||
| JP2020500162A5 (enExample) | ||
| JP2021502085A5 (enExample) | ||
| JP2018525979A5 (enExample) | ||
| JP2020505044A5 (enExample) | ||
| Lebek et al. | CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model | |
| King et al. | Correction of airway stem cells: genome editing approaches for the treatment of cystic fibrosis | |
| JP2017532955A5 (enExample) | ||
| Colella et al. | Gene therapy for Pompe disease: the time is now | |
| JP2015533786A5 (enExample) | ||
| JP2017511135A5 (enExample) | ||
| JP2017538401A5 (enExample) | ||
| Nam et al. | Intraperitoneal infusion of mesenchymal stem cell attenuates severity of collagen antibody induced arthritis | |
| Mathä et al. | Migration of lung resident group 2 innate lymphoid cells link allergic lung inflammation and liver immunity |