JP2016518142A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2016518142A5 JP2016518142A5 JP2016513016A JP2016513016A JP2016518142A5 JP 2016518142 A5 JP2016518142 A5 JP 2016518142A5 JP 2016513016 A JP2016513016 A JP 2016513016A JP 2016513016 A JP2016513016 A JP 2016513016A JP 2016518142 A5 JP2016518142 A5 JP 2016518142A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- dna
- combination
- sequence
- composition
- 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.)
- Pending
Links
- 210000004027 cell Anatomy 0.000 claims description 49
- 101710163270 Nuclease Proteins 0.000 claims description 37
- 108020004414 DNA Proteins 0.000 claims description 35
- 108090000623 proteins and genes Proteins 0.000 claims description 34
- 108091033319 polynucleotide Proteins 0.000 claims description 14
- 102000040430 polynucleotide Human genes 0.000 claims description 14
- 239000002157 polynucleotide Substances 0.000 claims description 14
- 230000010354 integration Effects 0.000 claims description 9
- 231100000331 toxic Toxicity 0.000 claims description 8
- 230000002588 toxic effect Effects 0.000 claims description 8
- 238000003776 cleavage reaction Methods 0.000 claims description 7
- 230000007017 scission Effects 0.000 claims description 7
- 108091026890 Coding region Proteins 0.000 claims description 6
- 239000013600 plasmid vector Substances 0.000 claims description 6
- 102000004169 proteins and genes Human genes 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 108700019146 Transgenes Proteins 0.000 claims description 5
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims description 5
- 239000013603 viral vector Substances 0.000 claims description 5
- 108091030071 RNAI Proteins 0.000 claims description 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 4
- 230000009368 gene silencing by RNA Effects 0.000 claims description 4
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 4
- -1 lymphokine Proteins 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 108091070501 miRNA Proteins 0.000 claims description 4
- 239000002679 microRNA Substances 0.000 claims description 4
- 238000012986 modification Methods 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 4
- 239000004055 small Interfering RNA Substances 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 3
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 108010001857 Cell Surface Receptors Proteins 0.000 claims description 2
- 102000004127 Cytokines Human genes 0.000 claims description 2
- 108090000695 Cytokines Proteins 0.000 claims description 2
- 108090000790 Enzymes Proteins 0.000 claims description 2
- 102000004190 Enzymes Human genes 0.000 claims description 2
- 102000007399 Nuclear hormone receptor Human genes 0.000 claims description 2
- 108020005497 Nuclear hormone receptor Proteins 0.000 claims description 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 claims description 2
- 239000000427 antigen Substances 0.000 claims description 2
- 108091007433 antigens Proteins 0.000 claims description 2
- 102000036639 antigens Human genes 0.000 claims description 2
- 239000003153 chemical reaction reagent Substances 0.000 claims description 2
- 239000003102 growth factor Substances 0.000 claims description 2
- 229940088597 hormone Drugs 0.000 claims description 2
- 239000005556 hormone Substances 0.000 claims description 2
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 102000006240 membrane receptors Human genes 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 230000001400 myeloablative effect Effects 0.000 claims description 2
- 108020004017 nuclear receptors Proteins 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 27
- 101800004910 Nuclease A Proteins 0.000 claims 2
- 101150090724 3 gene Proteins 0.000 claims 1
- 210000001185 bone marrow Anatomy 0.000 claims 1
- 230000001419 dependent effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 22
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 238000010322 bone marrow transplantation Methods 0.000 description 1
- 239000013604 expression vector Substances 0.000 description 1
- 230000009261 transgenic effect Effects 0.000 description 1
- 239000013598 vector Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361821872P | 2013-05-10 | 2013-05-10 | |
| US61/821,872 | 2013-05-10 | ||
| PCT/US2014/036972 WO2014182700A1 (en) | 2013-05-10 | 2014-05-06 | Delivery methods and compositions for nuclease-mediated genome engineering |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016518142A JP2016518142A (ja) | 2016-06-23 |
| JP2016518142A5 true JP2016518142A5 (enExample) | 2017-03-23 |
Family
ID=51864932
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016513016A Pending JP2016518142A (ja) | 2013-05-10 | 2014-05-06 | ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US10604771B2 (enExample) |
| EP (1) | EP2994531B1 (enExample) |
| JP (1) | JP2016518142A (enExample) |
| AU (1) | AU2014262867B2 (enExample) |
| CA (1) | CA2910427C (enExample) |
| WO (1) | WO2014182700A1 (enExample) |
Families Citing this family (117)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| PT3363902T (pt) | 2012-12-06 | 2019-12-19 | Sigma Aldrich Co Llc | Modificação e regulação de genoma baseadas em crispr |
| KR102874079B1 (ko) | 2013-03-15 | 2025-10-22 | 더 제너럴 하스피탈 코포레이션 | Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용 |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| AU2014265331B2 (en) | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| US10011850B2 (en) | 2013-06-21 | 2018-07-03 | The General Hospital Corporation | Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing |
| 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 |
| WO2015031619A1 (en) | 2013-08-28 | 2015-03-05 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| EP3988649B1 (en) | 2013-09-18 | 2024-11-27 | Kymab Limited | Methods, cells and organisms |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP3156493B1 (en) | 2014-04-30 | 2020-05-06 | Tsinghua University | Use of tale transcriptional repressor for modular construction of synthetic gene line in mammalian cell |
| WO2015165276A1 (zh) * | 2014-04-30 | 2015-11-05 | 清华大学 | 利用tale转录抑制子在哺乳动物细胞中模块化构建合成基因线路的试剂盒 |
| EP3152319A4 (en) | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| IL286474B2 (en) | 2014-06-23 | 2023-11-01 | Massachusetts Gen Hospital | Genome-wide random identification of DSBS assessed by sequencing (guide-sequence) |
| US9757420B2 (en) | 2014-07-25 | 2017-09-12 | Sangamo Therapeutics, Inc. | Gene editing for HIV gene therapy |
| US9816074B2 (en) | 2014-07-25 | 2017-11-14 | Sangamo Therapeutics, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US9616090B2 (en) | 2014-07-30 | 2017-04-11 | Sangamo Biosciences, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| WO2016114972A1 (en) | 2015-01-12 | 2016-07-21 | The Regents Of The University Of California | Heterodimeric cas9 and methods of use thereof |
| WO2016123243A1 (en) | 2015-01-28 | 2016-08-04 | The Regents Of The University Of California | Methods and compositions for labeling a single-stranded target nucleic acid |
| KR102598856B1 (ko) | 2015-03-03 | 2023-11-07 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| EP4600366A3 (en) * | 2015-03-31 | 2025-10-22 | SOHM, Inc. | Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell |
| EP3303634B1 (en) | 2015-06-03 | 2023-08-30 | The Regents of The University of California | Cas9 variants and methods of use thereof |
| AU2016316845B2 (en) | 2015-08-28 | 2022-03-10 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9512446B1 (en) | 2015-08-28 | 2016-12-06 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| US9926546B2 (en) | 2015-08-28 | 2018-03-27 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases |
| EP3347467B1 (en) | 2015-09-11 | 2021-06-23 | The General Hospital Corporation | Full interrogation of nuclease dsbs and sequencing (find-seq) |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| JP2018529353A (ja) | 2015-09-30 | 2018-10-11 | ザ ジェネラル ホスピタル コーポレイション | 配列決定法による切断事象の包括的生体外報告（ＣＩＲＣＬＥ−ｓｅｑ） |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| US10724020B2 (en) | 2016-02-02 | 2020-07-28 | Sangamo Therapeutics, Inc. | Compositions for linking DNA-binding domains and cleavage domains |
| EP3417061B1 (en) | 2016-02-18 | 2022-10-26 | The Regents of the University of California | Methods and compositions for gene editing in stem cells |
| CN109414414A (zh) | 2016-03-16 | 2019-03-01 | 戴维·格拉德斯通研究所 | 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物 |
| WO2017201476A1 (en) | 2016-05-20 | 2017-11-23 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| BR112019001783A2 (pt) | 2016-07-29 | 2019-05-07 | Regeneron Pharmaceuticals, Inc. | mamífero não humano, e, métodos para produzir o mamífero não humano e de triagem de um composto. |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| 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 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CN110023494A (zh) | 2016-09-30 | 2019-07-16 | 加利福尼亚大学董事会 | Rna指导的核酸修饰酶及其使用方法 |
| US10669539B2 (en) | 2016-10-06 | 2020-06-02 | Pioneer Biolabs, Llc | Methods and compositions for generating CRISPR guide RNA libraries |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| AU2017345430B2 (en) | 2016-10-20 | 2024-09-05 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
| JP7108608B2 (ja) | 2016-10-31 | 2022-07-28 | サンガモ セラピューティクス， インコーポレイテッド | 造血幹細胞および前駆細胞におけるｓｃｉｄ関連遺伝子の遺伝子修正 |
| CN110637090A (zh) * | 2016-11-02 | 2019-12-31 | D·基维利希 | 用于表达大型核酸转基因的质粒载体 |
| JP7317706B2 (ja) | 2016-12-14 | 2023-07-31 | リガンダル インコーポレイテッド | 核酸およびタンパク質ペイロード送達のための方法および組成物 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| SG10202109874VA (en) | 2017-01-19 | 2021-10-28 | Open Monoclonal Tech Inc | Human antibodies from transgenic rodents with multiple heavy chain immunoglobulin loci |
| KR102712656B1 (ko) | 2017-01-23 | 2024-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| US20190365818A1 (en) * | 2017-02-09 | 2019-12-05 | Allen Institute | Genetically-tagged stem cell lines and methods of use |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| EP3612551B1 (en) | 2017-04-21 | 2024-09-04 | The General Hospital Corporation | Variants of cpf1 (cas12a) with altered pam specificity |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| WO2018218188A2 (en) | 2017-05-25 | 2018-11-29 | The General Hospital Corporation | Base editors with improved precision and specificity |
| 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 |
| EP3672612A4 (en) | 2017-08-23 | 2021-09-29 | The General Hospital Corporation | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES WITH MODIFIED PAM SPECIFICITY |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| EP3694993A4 (en) | 2017-10-11 | 2021-10-13 | The General Hospital Corporation | SITE-SPECIFIC AND PARASITIC GENOMIC DESAMINATION DETECTION METHODS INDUCED BY BASIC EDITING TECHNOLOGIES |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| CN111565730B (zh) | 2017-11-09 | 2024-09-17 | 桑格摩生物治疗股份有限公司 | 细胞因子诱导型含sh2蛋白（cish）基因的遗传修饰 |
| US20220290186A1 (en) * | 2017-12-06 | 2022-09-15 | Generation Bio Co. | Gene editing using a modified closed-ended dna (cedna) |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019157239A1 (en) * | 2018-02-08 | 2019-08-15 | David Kiewlich | Plasmid vectors for expression of large nucleic acid transgenes |
| CN117757842A (zh) * | 2018-03-15 | 2024-03-26 | 星际治疗有限公司 | 合成dna运载体及使用方法 |
| EP3781585A4 (en) | 2018-04-17 | 2022-01-26 | The General Hospital Corporation | SENSITIVE IN VITRO ASSAYS FOR SUBSTRATE AND SITE PREFERENCES OF NUCLEIC ACID BINDERS, MODIFIERS AND CLEAVATORS |
| WO2019204457A1 (en) | 2018-04-18 | 2019-10-24 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (htt) |
| WO2019217354A1 (en) | 2018-05-07 | 2019-11-14 | Pioneer Hi-Bred International, Inc. | Methods and compositions for homology directed repair of double strand breaks in plant cell genomes |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US20210285009A1 (en) | 2018-07-13 | 2021-09-16 | The Regents Of The University Of California | Retrotransposon-based delivery vehicle and methods of use thereof |
| EP3841205A4 (en) | 2018-08-22 | 2022-08-17 | The Regents of The University of California | Variant type v crispr/cas effector polypeptides and methods of use thereof |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| US11407995B1 (en) | 2018-10-26 | 2022-08-09 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US11434477B1 (en) | 2018-11-02 | 2022-09-06 | Inari Agriculture Technology, Inc. | RNA-guided nucleases and DNA binding proteins |
| CA3117730A1 (en) | 2018-11-09 | 2020-05-14 | Inari Agriculture, Inc. | Rna-guided nucleases and dna binding proteins |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020163396A1 (en) | 2019-02-04 | 2020-08-13 | The General Hospital Corporation | Adenine dna base editor variants with reduced off-target rna editing |
| JP2022519410A (ja) | 2019-02-06 | 2022-03-24 | 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ｉ型の処置のための方法 |
| US12460230B2 (en) | 2019-02-10 | 2025-11-04 | The J. David Gladstone Institutes | Modified mitochondrion and methods of use thereof |
| CN113811607A (zh) | 2019-03-07 | 2021-12-17 | 加利福尼亚大学董事会 | CRISPR-Cas效应子多肽和其使用方法 |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| SG11202110607WA (en) | 2019-04-01 | 2021-10-28 | Tenaya Therapeutics Inc | Adeno-associated virus with engineered capsid |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US20220364123A1 (en) * | 2019-04-24 | 2022-11-17 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use |
| SG11202111256XA (en) | 2019-06-07 | 2021-11-29 | Regeneron Pharma | Non-human animals comprising a humanized albumin locus |
| WO2020264139A1 (en) * | 2019-06-26 | 2020-12-30 | Virovek, Inc. | Baculovirus expression system |
| WO2021030738A1 (en) * | 2019-08-14 | 2021-02-18 | Pairwise Plants Services, Inc. | Alteration of flavor traits in consumer crops via disablement of the myrosinase/glucosinolate system |
| JP2022549138A (ja) | 2019-09-18 | 2022-11-24 | インターガラクティック セラピューティクス インコーポレイテッド | 合成ｄｎａベクターおよびその使用法 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US12264341B2 (en) | 2020-01-24 | 2025-04-01 | The General Hospital Corporation | CRISPR-Cas enzymes with enhanced on-target activity |
| EP4093864A4 (en) | 2020-01-24 | 2024-04-10 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| EP4097144A4 (en) * | 2020-01-28 | 2024-02-28 | University of Southern California | GENOMIC ENGINEERING OF HUMAN IMMUNOGLOBULIN LOCUS TO EXPRESS RECOMBINANT BINDING-SITE MOLECULES |
| WO2021178556A1 (en) | 2020-03-04 | 2021-09-10 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for sensitization of tumor cells to immune therapy |
| JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート，インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
| US20240392272A1 (en) | 2021-09-28 | 2024-11-28 | Acrigen Biosciences | Compositions and methods for nucleic acid modifications |
| WO2023081756A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
| US20230279442A1 (en) | 2021-12-15 | 2023-09-07 | Versitech Limited | Engineered cas9-nucleases and method of use thereof |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| US12171178B2 (en) | 2022-07-18 | 2024-12-24 | Pairwise Plants Services, Inc. | Mustard green plants named ‘PWRG-1’, ‘PWRG-2,’ and ‘PWSGC’ |
| MA71557A (fr) | 2022-07-18 | 2025-05-30 | Renagade Therapeutics Management Inc. | Composants d'édition génique, systèmes et procédés d'utilisation |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025081042A1 (en) | 2023-10-12 | 2025-04-17 | Renagade Therapeutics Management Inc. | Nickase-retron template-based precision editing system and methods of use |
| WO2025155753A2 (en) | 2024-01-17 | 2025-07-24 | Renagade Therapeutics Management Inc. | Improved gene editing system, guides, and methods |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP0781331B1 (en) | 1994-08-20 | 2008-09-03 | Gendaq Limited | Improvements in or relating to 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 |
| US7364894B2 (en) * | 1995-09-15 | 2008-04-29 | Centelion | Circular DNA molecule having a conditional origin of replication, process for their preparation and their use in gene therapy |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| 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 |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| WO1999045132A1 (en) | 1998-03-02 | 1999-09-10 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| 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 |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | 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 |
| AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| 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 |
| WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| US7029847B2 (en) | 2000-05-16 | 2006-04-18 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| 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 |
| WO2003087341A2 (en) * | 2002-01-23 | 2003-10-23 | 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 |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| JP2006502748A (ja) | 2002-09-05 | 2006-01-26 | カリフォルニア インスティテュート オブ テクノロジー | 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法 |
| 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 |
| CA2562193A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| AU2005287278B2 (en) | 2004-09-16 | 2011-08-04 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity 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 |
| EP2213731B1 (en) | 2006-05-25 | 2013-12-04 | Sangamo BioSciences, Inc. | Variant foki cleavage half-domains |
| EP2447279B1 (en) | 2006-05-25 | 2014-04-09 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| CA2667414C (en) * | 2006-11-13 | 2015-12-29 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the human glucocorticoid receptor locus |
| US8110379B2 (en) * | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| EP2188384B1 (en) | 2007-09-27 | 2015-07-15 | Sangamo BioSciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| KR20160015400A (ko) | 2008-08-22 | 2016-02-12 | 상가모 바이오사이언스 인코포레이티드 | 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물 |
| 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 |
| SI2370575T1 (en) | 2008-12-17 | 2018-07-31 | Dow Agrosciences, Llc | Targeted integration in the Zp15 locus |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| CA2749440A1 (en) | 2009-01-15 | 2011-08-12 | The New Zealand Institute For Plant And Food Research Limited | Plant transformation using dna minicircles |
| CA2755192C (en) | 2009-03-20 | 2018-09-11 | Sangamo Biosciences, Inc. | Modification of cxcr4 using engineered zinc finger proteins |
| US9834787B2 (en) * | 2009-04-09 | 2017-12-05 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
| JP2013500018A (ja) * | 2009-07-24 | 2013-01-07 | 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 | ゲノム編集のための方法 |
| KR20120094166A (ko) | 2009-10-16 | 2012-08-23 | 베일러 칼리지 오브 메디신 | 유전자 치료 적용을 위한 초나선화된 미니서클 ｄｎａ |
| ES2751916T3 (es) * | 2010-02-08 | 2020-04-02 | Sangamo Therapeutics Inc | Semidominios de escisión genomanipulados |
| WO2011100058A1 (en) | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| CN103097527A (zh) * | 2010-02-26 | 2013-05-08 | 塞勒克提斯公司 | 用于将转基因插入安全港座位的内切核酸酶的用途 |
| US9567573B2 (en) | 2010-04-26 | 2017-02-14 | Sangamo Biosciences, Inc. | Genome editing of a Rosa locus using nucleases |
| CA2798988C (en) | 2010-05-17 | 2020-03-10 | Sangamo Biosciences, Inc. | Tal-effector (tale) dna-binding polypeptides and uses thereof |
| WO2012015938A2 (en) * | 2010-07-27 | 2012-02-02 | The Johns Hopkins University | Obligate heterodimer variants of foki cleavage domain |
| GB201014169D0 (en) * | 2010-08-25 | 2010-10-06 | Cambridge Entpr Ltd | In vitro hepatic differentiation |
| EP3450563A1 (en) * | 2011-02-25 | 2019-03-06 | Recombinetics, Inc. | Genetically modified animals and methods for making the same |
| CA3186126A1 (en) * | 2011-09-21 | 2013-03-28 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| CA2852955C (en) | 2011-10-27 | 2021-02-16 | Sangamo Biosciences, Inc. | Methods and compositions for modification of the hprt locus |
| FI3597749T3 (fi) * | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| KR20150027756A (ko) * | 2012-05-30 | 2015-03-12 | 베일러 칼리지 오브 메디신 | Dna 수복, 변경 및 대체를 위한 도구로서의 초나선 미니벡터 |
| AU2013355327A1 (en) * | 2012-12-05 | 2015-06-11 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of metabolic disorders |
-
2014
- 2014-05-06 EP EP14794094.4A patent/EP2994531B1/en active Active
- 2014-05-06 CA CA2910427A patent/CA2910427C/en active Active
- 2014-05-06 JP JP2016513016A patent/JP2016518142A/ja active Pending
- 2014-05-06 WO PCT/US2014/036972 patent/WO2014182700A1/en not_active Ceased
- 2014-05-06 US US14/271,008 patent/US10604771B2/en active Active
- 2014-05-06 AU AU2014262867A patent/AU2014262867B2/en not_active Ceased
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016518142A5 (enExample) | ||
| Slattery et al. | An expanded plasmid-based genetic toolbox enables Cas9 genome editing and stable maintenance of synthetic pathways in Phaeodactylum tricornutum | |
| CN107794272B (zh) | 一种高特异性的crispr基因组编辑体系 | |
| JP2015533786A5 (enExample) | ||
| US10858662B2 (en) | Genome editing with split Cas9 expressed from two vectors | |
| CN109913495B (zh) | 大鼠的遗传修饰 | |
| US20170009242A1 (en) | CRISPR-Mediated Genome Engineering for Protein Depletion | |
| US20200407693A1 (en) | Method for producing low-antigenic cell | |
| JP2018518181A5 (enExample) | ||
| RU2016126989A (ru) | Способы и композиции для направленной модификации генома | |
| CN106701830A (zh) | 一种敲除猪胚胎p66shc基因的方法 | |
| IL295858B2 (en) | gRNA molecules including TRACR and crRNA, compositions containing them and their use | |
| RU2014128987A (ru) | Быстрый способ клонирования и экспрессии сегментов гена родственной вариабельной области антитела | |
| JP2020519304A5 (enExample) | ||
| KR20210102309A (ko) | 유전적으로-변형된 세포를 제작하는 줄어든, 그리고 최소한의 조작 | |
| US20200318140A1 (en) | Methods and compositions for increasing rna activity in a cell | |
| AU2015233347A1 (en) | Engineering mammalian genome using DNA-guided Argonaute interference systems (DAIS) | |
| AU2021369494A1 (en) | Safe harbor loci | |
| CN110229816B (zh) | 用于敲除RBP4基因的sgRNA、RBP4基因缺失细胞株的构建方法及应用 | |
| Dong et al. | Cas12a/Cpf1 knock-in mice enable efficient multiplexed immune cell engineering | |
| CN102363792A (zh) | 制备IGF2b转基因鱼的慢病毒方法 | |
| Suda et al. | Molecular cytogenetic characterization of telomere‐specific repetitive DNA sequences in Rana rugosa | |
| US20250222103A1 (en) | Immune cells with combination gene perturbations | |
| KR20240011184A (ko) | Ciita 표적화 아연 핑거 뉴클레아제 | |
| Yu et al. | Behavior of calf Sertoli cells and fibroblast cells transfected with the human HNP-1 gene |