JP2017504354A5 - - Google Patents
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- JP2017504354A5 JP2017504354A5 JP2016557544A JP2016557544A JP2017504354A5 JP 2017504354 A5 JP2017504354 A5 JP 2017504354A5 JP 2016557544 A JP2016557544 A JP 2016557544A JP 2016557544 A JP2016557544 A JP 2016557544A JP 2017504354 A5 JP2017504354 A5 JP 2017504354A5
- Authority
- JP
- Japan
- Prior art keywords
- zinc finger
- pharmaceutical composition
- vector
- protein
- aav 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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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/US2014/069307 WO2015089046A1 (en) | 2013-12-09 | 2014-12-09 | Methods and compositions for treating hemophilia |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017504354A JP2017504354A (ja) | 2017-02-09 |
| JP2017504354A5 true JP2017504354A5 (enExample) | 2017-12-21 |
| JP6642943B2 JP6642943B2 (ja) | 2020-02-12 |
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 Before (1)
| 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 |
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) | US10081661B2 (enExample) |
| EP (3) | EP3080274B1 (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) | IL245778B (enExample) |
| WO (2) | WO2015089077A2 (enExample) |
Families Citing this family (98)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| EP4372090A3 (en) | 2013-11-07 | 2024-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| CA3229275A1 (en) * | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| WO2015127439A1 (en) | 2014-02-24 | 2015-08-27 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | 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 |
| WO2017011519A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| PE20181206A1 (es) * | 2015-10-28 | 2018-07-23 | Sangamo Therapeutics Inc | Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos |
| AU2016361350B2 (en) | 2015-11-23 | 2023-04-06 | Sangamo Therapeutics, Inc. | Methods and compositions for engineering immunity |
| WO2017106528A2 (en) | 2015-12-18 | 2017-06-22 | Sangamo Biosciences, Inc. | Targeted disruption of the t cell receptor |
| IL259935B2 (en) | 2015-12-18 | 2023-03-01 | Sangamo Therapeutics Inc | Directed cleavage of cell mhc receptor |
| BR112018014288A2 (pt) * | 2016-01-15 | 2018-12-18 | Univ Minnesota | métodos e composições para o tratamento de doença neurológica |
| CA3011049A1 (en) | 2016-02-02 | 2017-08-10 | Sangamo Therapeutics, Inc. | Compositions for linking dna-binding domains and cleavage 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 |
| CA3032699A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harvard College | Adenosine nucleobase editors and uses thereof |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| MY193234A (en) | 2016-08-24 | 2022-09-27 | Sangamo Therapeutics Inc | Regulation of gene expression using engineered nucleases |
| KR20240144493A (ko) | 2016-08-24 | 2024-10-02 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
| WO2018049009A2 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| US11306324B2 (en) | 2016-10-14 | 2022-04-19 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| CA3039673A1 (en) * | 2016-10-20 | 2018-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| JP2020500539A (ja) * | 2016-12-09 | 2020-01-16 | サンガモ セラピューティクス， インコーポレイテッド | 標的特異的ヌクレアーゼの送達 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| WO2018126087A1 (en) * | 2016-12-29 | 2018-07-05 | Applied Stemcell, 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 |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | 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 | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| EP3601562A1 (en) | 2017-03-23 | 2020-02-05 | President and Fellows of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| IL301115A (en) | 2017-04-28 | 2023-05-01 | Acuitas Therapeutics Inc | Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| US11655275B2 (en) | 2017-05-03 | 2023-05-23 | 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 |
| US11667934B2 (en) | 2017-06-15 | 2023-06-06 | 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 |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| JP2021500036A (ja) | 2017-10-16 | 2021-01-07 | ザ ブロード インスティテュート, インコーポレーテッドThe Broad Institute, Inc. | アデノシン塩基編集因子の使用 |
| KR102728416B1 (ko) * | 2017-10-17 | 2024-11-11 | 크리스퍼 테라퓨틱스 아게 | A형 혈우병을 위한 유전자 편집용 조성물 및 방법 |
| 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 |
| MA51637A (fr) * | 2018-01-12 | 2020-11-18 | Bayer Healthcare Llc | Compositions et méthodes pour l'édition génique par ciblage de la transferrine |
| JP7275152B2 (ja) * | 2018-02-08 | 2023-05-17 | 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ヌクレアーゼ |
| WO2019161310A1 (en) * | 2018-02-16 | 2019-08-22 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing by targeting fibrinogen-alpha |
| CA3096274A1 (en) | 2018-04-06 | 2019-10-10 | 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 |
| EP3829650A4 (en) | 2018-08-03 | 2022-05-04 | Sangamo Therapeutics, Inc. | CLINICAL PARAMETERS IMPROVED BY FACTOR VIII EXPRESSION |
| JP7432581B2 (ja) * | 2018-08-07 | 2024-02-16 | サンガモ セラピューティクス， インコーポレイテッド | ムコ多糖症ｉｉ型の処置のための方法 |
| 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 |
| AU2019325609A1 (en) | 2018-08-24 | 2021-03-18 | Csl Behring Gene Therapy, Inc. | Vector production in serum free media |
| EP4234570A3 (en) | 2018-09-18 | 2023-12-13 | 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. |
| IL322436A (en) | 2018-09-21 | 2025-09-01 | Acuitas Therapeutics Inc | Systems and methods for producing lipid nanoparticles and liposomes |
| SG11202103917VA (en) | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
| MX2021004455A (es) * | 2018-10-17 | 2021-08-11 | Crispr Therapeutics Ag | Composiciones y métodos para administrar transgenes. |
| WO2020082046A2 (en) * | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| MY208564A (en) * | 2018-10-18 | 2025-05-15 | Regeneron Pharma | Compositions and methods for transgene expression from an albumin locus |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CN113474328B (zh) | 2019-01-11 | 2025-07-11 | 爱康泰生治疗公司 | 用于脂质纳米颗粒递送活性剂的脂质 |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN112805026A (zh) * | 2019-02-06 | 2021-05-14 | 桑格摩生物治疗股份有限公司 | 用于治疗i型黏多糖贮积症的方法 |
| EP3923992A1 (en) | 2019-02-15 | 2021-12-22 | CRISPR Therapeutics AG | Gene editing for hemophilia a with improved factor viii expression |
| KR20210143230A (ko) | 2019-03-19 | 2021-11-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴클레오티드 서열을 편집하기 위한 방법 및 조성물 |
| AU2020253362A1 (en) | 2019-04-02 | 2021-11-04 | Sangamo Therapeutics, Inc. | Methods for the treatment of beta-thalassemia |
| MX2021011956A (es) * | 2019-04-03 | 2021-12-15 | Regeneron Pharma | Metodos y composiciones para la insercion de secuencias codificantes de anticuerpos en un locus de puerto seguro. |
| 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 |
| US12509703B2 (en) | 2019-05-21 | 2025-12-30 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| 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 |
| EP4146797A1 (en) | 2020-05-06 | 2023-03-15 | Orchard Therapeutics (Europe) Limited | Treatment for neurodegenerative diseases |
| BR112022022603A2 (pt) | 2020-05-08 | 2023-01-17 | Broad Inst Inc | Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla |
| AU2021308681A1 (en) | 2020-07-16 | 2023-03-09 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
| WO2022046760A2 (en) | 2020-08-25 | 2022-03-03 | 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 |
| MX2024005124A (es) * | 2021-10-27 | 2024-05-16 | Regeneron Pharma | Composiciones y metodos para expresar el factor ix para terapia para la hemofilia b. |
| CN118647600A (zh) | 2021-12-16 | 2024-09-13 | 爱康泰生治疗公司 | 用于脂质纳米颗粒制剂的脂质 |
| AU2023205708A1 (en) | 2022-01-07 | 2024-07-25 | Toolgen Incorporated | Method for predicting possible off-targets in gene editing process |
| US20250295810A1 (en) | 2022-05-09 | 2025-09-25 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified capsid for gene therapy |
| WO2023220035A1 (en) | 2022-05-09 | 2023-11-16 | Synteny Therapeutics, Inc. | Erythroparvovirus compositions and methods for gene therapy |
| EP4522210A1 (en) | 2022-05-09 | 2025-03-19 | Synteny Therapeutics, Inc. | Erythroparvovirus with a modified genome for gene therapy |
| US12383615B2 (en) | 2023-03-23 | 2025-08-12 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods |
| WO2024196965A1 (en) | 2023-03-23 | 2024-09-26 | 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 |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| AU704601B2 (en) | 1994-01-18 | 1999-04-29 | Scripps Research Institute, The | 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 |
| AU696455C (en) | 1994-03-23 | 2006-03-02 | 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 |
| EP0781331B1 (en) | 1994-08-20 | 2008-09-03 | 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 |
| WO1998024479A1 (en) | 1996-12-02 | 1998-06-11 | 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 |
| WO1999029848A1 (en) | 1997-12-05 | 1999-06-17 | 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 |
| AU746454B2 (en) | 1998-03-02 | 2002-05-02 | 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 |
| 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 |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| 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 |
| CA2398590C (en) | 2000-02-08 | 2012-08-28 | 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 |
| HU229101B1 (en) | 2000-04-28 | 2013-07-29 | St Jude Childrens Res Hospital | Dna transfection system for the generation of infectious influenza 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 |
| US20030232410A1 (en) | 2002-03-21 | 2003-12-18 | Monika Liljedahl | 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 |
| US9447434B2 (en) | 2002-09-05 | 2016-09-20 | 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 |
| 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 |
| 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 |
| US20080131962A1 (en) | 2006-05-25 | 2008-06-05 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | 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 |
| WO2007014275A2 (en) | 2005-07-26 | 2007-02-01 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| CA3055968A1 (en) | 2005-10-18 | 2007-04-26 | Duke University | Rationally-designed meganucleases with altered sequence specificity and dna-binding affinity |
| US8129510B2 (en) | 2006-03-30 | 2012-03-06 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
| US7951925B2 (en) | 2006-05-25 | 2011-05-31 | 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 |
| CN101679977B (zh) | 2007-04-26 | 2013-05-01 | 桑格摩生物科学股份有限公司 | 向ppp1r12c基因座的靶向整合 |
| CN101855231B (zh) | 2007-06-19 | 2014-06-11 | 路易斯安那州州立大学及农业机械学院管理委员会 | 信使rna帽的抗-反向硫代磷酸类似物的合成和用途 |
| US8790345B2 (en) | 2007-08-21 | 2014-07-29 | Zimmer, Inc. | Titanium alloy with oxidized zirconium for a prosthetic implant |
| CA2700231C (en) | 2007-09-27 | 2018-09-18 | 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 |
| CA2722238C (en) | 2008-04-22 | 2017-11-28 | Life Sciences Research Partners Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| EP2313515B1 (en) | 2008-08-22 | 2015-03-04 | Sangamo BioSciences, Inc. | Methods and compositions for targeted single-stranded cleavage and targeted integration |
| EP2352369B1 (en) | 2008-12-04 | 2017-04-26 | Sangamo BioSciences, Inc. | Genome editing in rats using zinc-finger nucleases |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| 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 |
| 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" |
| WO2011097036A1 (en) | 2010-02-08 | 2011-08-11 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
| WO2011100058A1 (en) | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| US8771985B2 (en) | 2010-04-26 | 2014-07-08 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using zinc-finger nucleases |
| EP2571512B1 (en) | 2010-05-17 | 2017-08-23 | Sangamo BioSciences, Inc. | Novel dna-binding proteins 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 |
| HUE029855T2 (en) | 2011-07-05 | 2017-04-28 | Bioasis Technologies Inc | p97 antibody conjugates |
| JP6185916B2 (ja) * | 2011-09-21 | 2017-08-23 | サンガモ セラピューティクス， インコーポレイテッド | 導入遺伝子発現を制御するための方法および組成物 |
| US9222105B2 (en) | 2011-10-27 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the HPRT locus |
| US10570378B2 (en) * | 2012-02-28 | 2020-02-25 | Sigma-Aldrich Co. Llc | Targeted histone acetylation |
| EP2847338B1 (en) * | 2012-05-07 | 2018-09-19 | Sangamo Therapeutics, 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 |
| JP6225182B2 (ja) | 2012-07-11 | 2017-11-01 | サンガモ セラピューティクス， インコーポレイテッド | リソソーム貯蔵疾患の処置のための方法および組成物 |
| CA2888931C (en) | 2012-10-26 | 2023-09-05 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| HK1223401A1 (zh) | 2013-05-15 | 2017-07-28 | 桑格摩生物科学股份有限公司 | 用於治疗遗传病状的方法和组合物 |
| US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | 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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