JP2018513682A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018513682A5 JP2018513682A5 JP2017552447A JP2017552447A JP2018513682A5 JP 2018513682 A5 JP2018513682 A5 JP 2018513682A5 JP 2017552447 A JP2017552447 A JP 2017552447A JP 2017552447 A JP2017552447 A JP 2017552447A JP 2018513682 A5 JP2018513682 A5 JP 2018513682A5
- Authority
- JP
- Japan
- Prior art keywords
- modified
- nucleotide sequence
- nucleotide
- nucleotides
- cells
- 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
- 125000003729 nucleotide group Chemical group 0.000 claims description 105
- 238000000034 method Methods 0.000 claims description 101
- 239000002773 nucleotide Substances 0.000 claims description 76
- 210000004027 cell Anatomy 0.000 claims description 35
- 229920001184 polypeptide Polymers 0.000 claims description 35
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 35
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 35
- 108020004707 nucleic acids Proteins 0.000 claims description 24
- 102000039446 nucleic acids Human genes 0.000 claims description 24
- 150000007523 nucleic acids Chemical class 0.000 claims description 24
- 108090000623 proteins and genes Proteins 0.000 claims description 22
- 108020004414 DNA Proteins 0.000 claims description 14
- 230000004048 modification Effects 0.000 claims description 13
- 238000012986 modification Methods 0.000 claims description 13
- 230000033228 biological regulation Effects 0.000 claims description 8
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims description 8
- 108091033409 CRISPR Proteins 0.000 claims description 6
- 230000035772 mutation Effects 0.000 claims description 6
- 210000000130 stem cell Anatomy 0.000 claims description 6
- 108020005004 Guide RNA Proteins 0.000 claims description 5
- 230000000694 effects Effects 0.000 claims description 5
- 108020004999 messenger RNA Proteins 0.000 claims description 5
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 4
- 210000002865 immune cell Anatomy 0.000 claims description 4
- 230000006780 non-homologous end joining Effects 0.000 claims description 4
- 210000005105 peripheral blood lymphocyte Anatomy 0.000 claims description 4
- 210000003819 peripheral blood mononuclear cell Anatomy 0.000 claims description 4
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims description 4
- 238000010354 CRISPR gene editing Methods 0.000 claims description 3
- 230000000295 complement effect Effects 0.000 claims description 3
- 239000013604 expression vector Substances 0.000 claims description 3
- 239000012634 fragment Substances 0.000 claims description 3
- 102000004169 proteins and genes Human genes 0.000 claims description 3
- 238000003259 recombinant expression Methods 0.000 claims description 3
- 125000004200 2-methoxyethyl group Chemical group [H]C([H])([H])OC([H])([H])C([H])([H])* 0.000 claims description 2
- 108010042407 Endonucleases Proteins 0.000 claims description 2
- 102000004533 Endonucleases Human genes 0.000 claims description 2
- 241000233866 Fungi Species 0.000 claims description 2
- 108091027544 Subgenomic mRNA Proteins 0.000 claims description 2
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 2
- 230000004913 activation Effects 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 claims description 2
- 238000004520 electroporation Methods 0.000 claims description 2
- 230000014509 gene expression Effects 0.000 claims description 2
- 238000010362 genome editing Methods 0.000 claims description 2
- 230000003394 haemopoietic effect Effects 0.000 claims description 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 2
- 230000001939 inductive effect Effects 0.000 claims description 2
- 230000005764 inhibitory process Effects 0.000 claims description 2
- 210000002901 mesenchymal stem cell Anatomy 0.000 claims description 2
- 210000001616 monocyte Anatomy 0.000 claims description 2
- 210000000822 natural killer cell Anatomy 0.000 claims description 2
- 210000001178 neural stem cell Anatomy 0.000 claims description 2
- 229940046166 oligodeoxynucleotide Drugs 0.000 claims description 2
- 210000000056 organ Anatomy 0.000 claims description 2
- 239000002243 precursor Substances 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 9
- 208000035475 disorder Diseases 0.000 description 6
- 208000026350 Inborn Genetic disease Diseases 0.000 description 3
- 201000010099 disease Diseases 0.000 description 3
- 208000016361 genetic disease Diseases 0.000 description 3
- 208000024172 Cardiovascular disease Diseases 0.000 description 2
- 208000019693 Lung disease Diseases 0.000 description 2
- 208000015294 blood coagulation disease Diseases 0.000 description 2
- 208000017169 kidney disease Diseases 0.000 description 2
- 208000019423 liver disease Diseases 0.000 description 2
- 208000024827 Alzheimer disease Diseases 0.000 description 1
- 206010003805 Autism Diseases 0.000 description 1
- 208000020706 Autistic disease Diseases 0.000 description 1
- 201000003883 Cystic fibrosis Diseases 0.000 description 1
- 208000001914 Fragile X syndrome Diseases 0.000 description 1
- 208000031886 HIV Infections Diseases 0.000 description 1
- 208000037357 HIV infectious disease Diseases 0.000 description 1
- 208000031220 Hemophilia Diseases 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 206010061218 Inflammation Diseases 0.000 description 1
- 206010028980 Neoplasm Diseases 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- 102000029797 Prion Human genes 0.000 description 1
- 108091000054 Prion Proteins 0.000 description 1
- 208000012827 T-B+ severe combined immunodeficiency due to gamma chain deficiency Diseases 0.000 description 1
- 208000002903 Thalassemia Diseases 0.000 description 1
- 208000023940 X-Linked Combined Immunodeficiency disease Diseases 0.000 description 1
- 201000007146 X-linked severe combined immunodeficiency Diseases 0.000 description 1
- 206010064930 age-related macular degeneration Diseases 0.000 description 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 208000016097 disease of metabolism Diseases 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 206010013663 drug dependence Diseases 0.000 description 1
- 208000030533 eye disease Diseases 0.000 description 1
- 208000033519 human immunodeficiency virus infectious disease Diseases 0.000 description 1
- 230000004054 inflammatory process Effects 0.000 description 1
- 201000006370 kidney failure Diseases 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 208000002780 macular degeneration Diseases 0.000 description 1
- 208000030159 metabolic disease Diseases 0.000 description 1
- 239000000693 micelle Substances 0.000 description 1
- 210000003205 muscle Anatomy 0.000 description 1
- 239000002105 nanoparticle Substances 0.000 description 1
- 230000001537 neural effect Effects 0.000 description 1
- 230000000926 neurological effect Effects 0.000 description 1
- 201000000980 schizophrenia Diseases 0.000 description 1
- 208000007056 sickle cell anemia Diseases 0.000 description 1
- 208000011117 substance-related disease Diseases 0.000 description 1
- 230000000699 topical effect Effects 0.000 description 1
- 239000000277 virosome Substances 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562143729P | 2015-04-06 | 2015-04-06 | |
| US62/143,729 | 2015-04-06 | ||
| US201562160545P | 2015-05-12 | 2015-05-12 | |
| US62/160,545 | 2015-05-12 | ||
| PCT/US2016/026028 WO2016164356A1 (en) | 2015-04-06 | 2016-04-05 | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018513682A JP2018513682A (ja) | 2018-05-31 |
| JP2018513682A5 true JP2018513682A5 (enExample) | 2019-05-16 |
| JP6873911B2 JP6873911B2 (ja) | 2021-05-19 |
Family
ID=55910340
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017552447A Active JP6873911B2 (ja) | 2015-04-06 | 2016-04-05 | 初代細胞において標的核酸の遺伝子調節を誘導するためにインビトロで行う方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US11306309B2 (enExample) |
| EP (1) | EP3280803B1 (enExample) |
| JP (1) | JP6873911B2 (enExample) |
| KR (2) | KR102648489B1 (enExample) |
| CN (2) | CN107787367B (enExample) |
| AU (2) | AU2016246450B2 (enExample) |
| CA (1) | CA2981715A1 (enExample) |
| ES (1) | ES2884838T3 (enExample) |
| WO (1) | WO2016164356A1 (enExample) |
Families Citing this family (166)
| 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 |
| 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 |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| WO2015153789A1 (en) * | 2014-04-01 | 2015-10-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1) |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US10040048B1 (en) | 2014-09-25 | 2018-08-07 | Synthego Corporation | Automated modular system and method for production of biopolymers |
| CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| JP2017538427A (ja) | 2014-12-18 | 2017-12-28 | インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド | Ｃｒｉｓｐｒ系組成物及び使用方法 |
| US12129471B2 (en) | 2015-02-23 | 2024-10-29 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| WO2016135558A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| CN107787367B (zh) | 2015-04-06 | 2021-10-26 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| US11279928B2 (en) | 2015-06-29 | 2022-03-22 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
| 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 |
| EP3159407A1 (en) * | 2015-10-23 | 2017-04-26 | Silence Therapeutics (London) Ltd | Guide rnas, methods and uses |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP3371306B8 (en) | 2015-11-04 | 2023-02-22 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| WO2017131237A1 (ja) * | 2016-01-30 | 2017-08-03 | 株式会社ボナック | 人工単一ガイドｒｎａ及びその用途 |
| WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
| KR101710026B1 (ko) | 2016-08-10 | 2017-02-27 | 주식회사 무진메디 | Cas9 단백질 및 가이드 RNA의 혼성체를 함유하는 나노 리포좀 전달체 조성물 |
| US12012598B2 (en) * | 2016-08-12 | 2024-06-18 | Toolgen Incorporated | Manipulated immunoregulatory element and immunity altered thereby |
| MX2019002004A (es) | 2016-08-19 | 2019-10-09 | Whitehead Inst Biomedical Res | Metodos para editar la metilacion del adn. |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018049075A1 (en) | 2016-09-07 | 2018-03-15 | Flagship Pioneering, Inc. | Methods and compositions for modulating gene expression |
| US11242542B2 (en) | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| WO2018068053A2 (en) | 2016-10-07 | 2018-04-12 | Integrated Dna Technologies, Inc. | S. pyogenes cas9 mutant genes and polypeptides encoded by same |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| CA3044101A1 (en) | 2016-11-22 | 2018-05-31 | Integrated Dna Technologies, Inc. | Crispr/cpf1 systems and methods |
| EP3551757A1 (en) | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
| WO2018111947A1 (en) | 2016-12-12 | 2018-06-21 | Integrated Dna Technologies, Inc. | Genome editing enhancement |
| WO2018111944A1 (en) | 2016-12-12 | 2018-06-21 | Whitehead Institute For Biomedical Research | Regulation of transcription through ctcf loop anchors |
| CN118460529A (zh) | 2016-12-20 | 2024-08-09 | 百时美施贵宝公司 | 提高细胞基因组中同源定向修复(hdr)效率的方法 |
| JP7383478B2 (ja) | 2016-12-22 | 2023-11-20 | インテリア セラピューティクス，インコーポレイテッド | アルファ１アンチトリプシン欠乏症を治療するための組成物および方法 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| CN110249052B (zh) * | 2016-12-30 | 2024-04-12 | 爱迪塔斯医药公司 | 合成的指导分子、组合物和与其相关的方法 |
| JOP20190166A1 (ar) * | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| US11873496B2 (en) | 2017-01-09 | 2024-01-16 | Whitehead Institute For Biomedical Research | Methods of altering gene expression by perturbing transcription factor multimers that structure regulatory loops |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| AU2018221730B2 (en) * | 2017-02-15 | 2024-06-20 | Novo Nordisk A/S | Donor repair templates multiplex genome editing |
| WO2018154413A1 (en) * | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Materials and methods for treatment of dystrophic epidermolysis bullosa (deb) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders |
| EP3585896A1 (en) * | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Materials and methods for treatment of merosin-deficient cogenital muscular dystrophy (mdcmd) and other laminin, alpha 2 (lama2) gene related conditions or disorders |
| KR102709603B1 (ko) | 2017-02-28 | 2024-09-27 | 보르 바이오파마 인크. | 계통 특이적 단백질의 억제를 위한 조성물 및 방법 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| 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 |
| WO2018175480A1 (en) * | 2017-03-20 | 2018-09-27 | The Research Foundation For The State University Of New York | Methods of inhibiting fungal ceramide synthase for treatment of cryptococcus neoformans infection |
| IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
| WO2018183808A1 (en) * | 2017-03-31 | 2018-10-04 | Agenovir Corporation | Antiviral therapeutic |
| US12275963B2 (en) | 2017-05-08 | 2025-04-15 | Toolgen Incorporated | Artificially manipulated immune cell |
| BR112019023608A2 (pt) | 2017-05-12 | 2020-05-26 | Crispr Therapeutics Ag | Materiais e métodos para células manipuladas e seus usos em imuno-oncologia |
| 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 |
| US11166985B2 (en) | 2017-05-12 | 2021-11-09 | Crispr Therapeutics Ag | Materials and methods for engineering cells and uses thereof in immuno-oncology |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| EP3681637A4 (en) | 2017-09-11 | 2021-06-02 | Synthego Corporation | SYSTEM AND PROCEDURE FOR BIOPOLYMER SYNTHESIS |
| EP3686275A4 (en) * | 2017-09-18 | 2021-09-29 | Edigene Inc. | GENE EDITING LYMPHOCYTE T AND RELATED USE |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| MA50849A (fr) | 2017-10-26 | 2020-09-02 | Vertex Pharma | Substances et procédés pour le traitement d'hémoglobinopathies |
| JP2021505587A (ja) | 2017-12-05 | 2021-02-18 | バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ | Ｃｒｉｓｐｒ−ｃａｓ９改変ｃｄ３４＋ヒト造血幹・前駆細胞及びその使用 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| KR102002443B1 (ko) | 2018-01-22 | 2019-07-23 | 경상대학교산학협력단 | 식물체에서 상동재조합 기반의 유전자 편집 효율을 증가시키는 방법 |
| US20190233816A1 (en) * | 2018-01-26 | 2019-08-01 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
| MA51787A (fr) | 2018-02-05 | 2020-12-16 | Vertex Pharma | Substances et méthodes de traitement d'hémoglobinopathies |
| EP3749768A1 (en) | 2018-02-05 | 2020-12-16 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| CN110229814B (zh) * | 2018-03-06 | 2025-11-14 | 中国科学院动物研究所 | 改进的向导rna |
| WO2019189573A1 (ja) * | 2018-03-29 | 2019-10-03 | 学校法人自治医科大学 | ゲノム編集方法、組成物、細胞、細胞製剤、及び細胞製剤の製造方法 |
| CA3096708A1 (en) * | 2018-04-18 | 2019-10-24 | Yale University | Compositions and methods for multiplexed tumor vaccination with endogenous gene activation |
| US20200063100A1 (en) | 2018-05-11 | 2020-02-27 | Crispr Therapeutics Ag | Methods and compositions for treating cancer |
| CN112654710A (zh) | 2018-05-16 | 2021-04-13 | 辛瑟高公司 | 用于指导rna设计和使用的方法和系统 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| CN108624592B (zh) * | 2018-05-24 | 2021-08-17 | 苏州大学张家港工业技术研究院 | 针对DJ-1基因编辑的sgRNA筛选及其载体与应用 |
| WO2019226976A1 (en) * | 2018-05-25 | 2019-11-28 | New York Institute Of Technology | Method and system for use in direct sequencing of rna |
| US12398360B2 (en) * | 2018-06-01 | 2025-08-26 | Avectas Limited | Cell engineering platform |
| JP7832761B2 (ja) * | 2018-06-08 | 2026-03-18 | インテリア セラピューティクス，インコーポレイテッド | 遺伝子編集のための修飾されたガイドｒｎａ |
| CN110616187B (zh) * | 2018-06-20 | 2021-12-03 | 西安桑尼赛尔生物医药有限公司 | CRISPR-Cas9高效敲入嵌合抗原受体基因到T细胞特定基因组位点的方法及应用 |
| CN110616233B (zh) * | 2018-06-20 | 2022-02-22 | 西安桑尼赛尔生物医药有限公司 | CRISPR-Cas9高效敲除原代T细胞基因的方法及其应用 |
| AU2019291918B2 (en) | 2018-06-29 | 2025-06-12 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| CA3110837A1 (en) * | 2018-08-28 | 2020-03-05 | Vor Biopharma Inc. | Genetically engineered hematopoietic stem cells and uses thereof |
| WO2020061591A1 (en) | 2018-09-21 | 2020-03-26 | President And Fellows Of Harvard College | Methods and compositions for treating diabetes, and methods for enriching mrna coding for secreted proteins |
| WO2020068643A1 (en) * | 2018-09-25 | 2020-04-02 | Excision Biotherapeutics, Inc. | CRISPRs WITH IMPROVED SPECIFICITY |
| KR102126573B1 (ko) | 2018-10-18 | 2020-06-26 | 대한민국 | CRISPR-Cas9 시스템을 이용한 유색고약버섯의 리그닌 분해효소의 유전자 편집방법 및 이의 용도 |
| CN111088253A (zh) * | 2018-10-24 | 2020-05-01 | 广州鼓润医疗科技有限公司 | 针对hbb-28地中海贫血基因的crispr单碱基供体修复体系 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CN111254164A (zh) * | 2018-11-30 | 2020-06-09 | 中国科学院大连化学物理研究所 | 一种快速建立crispr基因编辑肝癌细胞株的方法及细胞株 |
| CN111321171A (zh) * | 2018-12-14 | 2020-06-23 | 江苏集萃药康生物科技有限公司 | 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法 |
| CN109652440A (zh) * | 2018-12-28 | 2019-04-19 | 北京市农林科学院 | VQRn-Cas9&PmCDA1&UGI碱基编辑系统在植物基因编辑中的应用 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN110157695A (zh) * | 2019-01-25 | 2019-08-23 | 南昌大学 | 一种植物RNA m5C甲基化修饰的系统及方法 |
| WO2020191241A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3138633A1 (en) | 2019-04-30 | 2020-11-05 | Crispr Therapeutics Ag | Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19 |
| AU2020280103A1 (en) | 2019-05-23 | 2021-12-23 | Vor Biopharma Inc. | Compositions and methods for CD33 modification |
| CN110283838B (zh) * | 2019-06-27 | 2022-03-25 | 安徽省农业科学院水稻研究所 | 一种高剪切效率ScCas9基因及其应用 |
| US12655438B2 (en) * | 2019-08-09 | 2026-06-16 | Regents Of The University Of Minnesota | Augmented sgRNAs and methods for their use to enhance somatic and germline plant genome engineering |
| WO2021041977A1 (en) | 2019-08-28 | 2021-03-04 | Vor Biopharma, Inc. | Compositions and methods for cd123 modification |
| AU2020338011A1 (en) | 2019-08-28 | 2022-03-10 | Vor Biopharma Inc. | Compositions and methods for CLL1 modification |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| JP7689949B2 (ja) | 2019-10-03 | 2025-06-09 | アーティサン ディベロップメント ラブズ インコーポレイテッド | 操作されたデュアルガイド核酸を有するｃｒｉｓｐｒシステム |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| EP4041916A4 (en) * | 2019-10-11 | 2023-11-01 | Blackhawk Genomics LLC | IDENTIFICATION OF GENOMIC STRUCTURAL VARIANTS USING LONG READ SEQUENCING |
| CA3158555A1 (en) | 2019-10-23 | 2021-04-29 | Pairwise Plants Services, Inc. | Compositions and methods for rna-templated editing in plants |
| JP7785002B2 (ja) * | 2019-11-05 | 2025-12-12 | ペアーワイズ プランツ サービシズ， インコーポレイテッド | 対立遺伝子のｒｎａにコードされたｄｎａの置換のための組成物および方法 |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| AU2020382219A1 (en) | 2019-11-13 | 2022-05-12 | Crispr Therapeutics Ag | Manufacturing process for making T cells expressing chimeric antigen receptors |
| AU2020401206A1 (en) * | 2019-12-11 | 2022-07-14 | Intellia Therapeutics, Inc. | Modified guide RNAs for gene editing |
| IL271656A (en) | 2019-12-22 | 2021-06-30 | Yeda Res & Dev | System and methods for identifying cells that have undergone genome editing |
| CA3177481A1 (en) | 2020-05-08 | 2021-11-11 | David R. Liu | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2021237117A1 (en) * | 2020-05-22 | 2021-11-25 | Insitro, Inc. | Predicting disease outcomes using machine learned models |
| CA3190447A1 (en) * | 2020-08-03 | 2022-02-10 | The Board Of Trustees Of The Leland Stanford Junior University | Gene correction for scid-x1 in long-term hematopoietic stem cells |
| CN112111507B (zh) * | 2020-08-10 | 2022-08-23 | 江苏大学 | 一种灰树花CRISPR-Cas9基因编辑系统、方法及应用 |
| CN112266911B (zh) * | 2020-08-27 | 2022-07-22 | 清华大学 | 核酸分子 |
| EP4204565A1 (en) | 2020-08-28 | 2023-07-05 | Vor Biopharma Inc. | Compositions and methods for cll1 modification |
| EP4204564A1 (en) | 2020-08-28 | 2023-07-05 | Vor Biopharma Inc. | Compositions and methods for cd123 modification |
| EP4211245A1 (en) | 2020-09-14 | 2023-07-19 | Vor Biopharma Inc. | Compositions and methods for cd5 modification |
| WO2022056489A1 (en) | 2020-09-14 | 2022-03-17 | Vor Biopharma, Inc. | Compositions and methods for cd38 modification |
| WO2022061115A1 (en) | 2020-09-18 | 2022-03-24 | Vor Biopharma Inc. | Compositions and methods for cd7 modification |
| WO2022067240A1 (en) | 2020-09-28 | 2022-03-31 | Vor Biopharma, Inc. | Compositions and methods for cd6 modification |
| WO2022072643A1 (en) | 2020-09-30 | 2022-04-07 | Vor Biopharma Inc. | Compositions and methods for cd30 gene modification |
| CN112126661B (zh) * | 2020-09-30 | 2022-07-22 | 上海中医药大学 | 一种高效敲除nk细胞中tigit基因的方法 |
| WO2022081585A1 (en) * | 2020-10-12 | 2022-04-21 | The Board Of Trustees Of The Leland Stanford Junior University | Gene correction for x-cgd in hematopoietic stem and progenitor cells |
| CA3199623A1 (en) | 2020-10-27 | 2022-05-05 | Vor Biopharma Inc. | Compositions and methods for treating hematopoietic malignancy |
| WO2022094245A1 (en) | 2020-10-30 | 2022-05-05 | Vor Biopharma, Inc. | Compositions and methods for bcma modification |
| CN112553195B (zh) * | 2020-11-05 | 2022-04-05 | 南方医科大学 | 一种用于CRISPR-Cas9定点突变编辑DNMT1基因的试剂及其应用 |
| WO2022104090A1 (en) | 2020-11-13 | 2022-05-19 | Vor Biopharma Inc. | Methods and compositions relating to genetically engineered cells expressing chimeric antigen receptors |
| AU2021413252A1 (en) | 2020-12-31 | 2023-06-08 | Vor Biopharma Inc. | Compositions and methods for cd34 gene modification |
| AU2022227650A1 (en) | 2021-02-25 | 2023-10-12 | Celyntra Therapeutics Sa | Compositions and methods for targeting, editing, or modifying genes |
| JP2024519513A (ja) | 2021-03-01 | 2024-05-15 | ニューヨーク・ゲノム・センター，インコーポレーテッド | 細胞トランスクリプトームのコンビナトリアル標的化のための方法及び組成物 |
| KR102809622B1 (ko) * | 2021-03-09 | 2025-05-19 | 일루미나, 인코포레이티드 | 세포에서 단백질-코딩 변이체의 발현 분석 |
| WO2022217086A1 (en) | 2021-04-09 | 2022-10-13 | Vor Biopharma Inc. | Photocleavable guide rnas and methods of use thereof |
| CN117616135A (zh) * | 2021-05-07 | 2024-02-27 | 布罗德研究所股份有限公司 | 亚细胞分辨率的时空分辨转录组学 |
| WO2022256448A2 (en) | 2021-06-01 | 2022-12-08 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing, or modifying genes |
| WO2022266139A2 (en) | 2021-06-14 | 2022-12-22 | Graphite Bio, Inc. | Methods to genetically engineer hematopoietic stem and progenitor cells for red cell specific expression of therapeutic proteins |
| CA3223311A1 (en) | 2021-06-18 | 2022-12-22 | Andrea BARGHETTI | Compositions and methods for targeting, editing or modifying human genes |
| WO2023283585A2 (en) | 2021-07-06 | 2023-01-12 | Vor Biopharma Inc. | Inhibitor oligonucleotides and methods of use thereof |
| EP4381062A1 (en) | 2021-08-02 | 2024-06-12 | Vor Biopharma Inc. | Compositions and methods for gene modification |
| WO2023039586A1 (en) * | 2021-09-10 | 2023-03-16 | Agilent Technologies, Inc. | Guide rnas with chemical modification for prime editing |
| EP4402266A4 (en) * | 2021-09-14 | 2025-11-05 | Agilent Technologies Inc | GUIDELINES FOR USING RNA WITH CHEMICAL MODIFICATIONS |
| EP4408991A2 (en) | 2021-09-27 | 2024-08-07 | Vor Biopharma Inc. | Fusion polypeptides for genetic editing and methods of use thereof |
| EP4413146A2 (en) * | 2021-10-05 | 2024-08-14 | The Board of Trustees of the Leland Stanford Junior University | Treatment of polycythemia vera via crispr/aav6 genome editing |
| EP4430186A1 (en) | 2021-11-09 | 2024-09-18 | Vor Biopharma Inc. | Compositions and methods for erm2 modification |
| JP2025501265A (ja) * | 2021-12-30 | 2025-01-17 | シロ ダイアグノスティクス インコーポレイテッド | 核酸の等温増幅のための組成物および方法 |
| WO2023155924A1 (en) * | 2022-02-21 | 2023-08-24 | Huidagene Therapeutics Co., Ltd. | Guide rna and uses thereof |
| US20250179531A1 (en) | 2022-02-25 | 2025-06-05 | Vor Biopharma Inc. | Compositions and methods for homology-directed repair gene modification |
| EP4486881A1 (en) | 2022-03-01 | 2025-01-08 | Celyntra Therapeutics SA | Composition and methods for transgene insertion |
| CN118974256A (zh) | 2022-03-29 | 2024-11-15 | 锐正基因(苏州)有限公司 | 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法 |
| US20250295695A1 (en) | 2022-04-04 | 2025-09-25 | Vor Biopharma Inc. | Compositions and methods for mediating epitope engineering |
| EP4526884A2 (en) | 2022-05-20 | 2025-03-26 | Artisan Development Labs, Inc. | Systems and methods for assessing risk of genome editing events |
| WO2024015925A2 (en) | 2022-07-13 | 2024-01-18 | Vor Biopharma Inc. | Compositions and methods for artificial protospacer adjacent motif (pam) generation |
| EP4590820A2 (en) | 2022-09-22 | 2025-07-30 | Accuredit Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease |
| WO2024062138A1 (en) | 2022-09-23 | 2024-03-28 | Mnemo Therapeutics | Immune cells comprising a modified suv39h1 gene |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| WO2024137533A1 (en) | 2022-12-19 | 2024-06-27 | Graphite Bio, Inc. | Improved peptide inhibitors of p53 binding protein 53bp1 |
| WO2024168312A1 (en) | 2023-02-09 | 2024-08-15 | Vor Biopharma Inc. | Methods for treating hematopoietic malignancy |
| WO2024193704A1 (en) * | 2023-03-22 | 2024-09-26 | Huidagene Therapeutics Co., Ltd. | Guide nucleic acids targeting dmd and uses thereof |
| JP2026523341A (ja) | 2023-05-15 | 2026-07-14 | エヌクロマ・バイオ，インコーポレーテッド | Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法 |
| WO2025030010A1 (en) | 2023-08-01 | 2025-02-06 | Vor Biopharma Inc. | Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof |
| WO2025177303A1 (en) * | 2024-02-19 | 2025-08-28 | Micro Crispr Pvt Ltd. | Crispr-mediated gene editing to induce fetal hemoglobin |
| CN118325904B (zh) * | 2024-06-17 | 2024-10-01 | 天海元祺生物科技(天津)有限公司 | 高度靶向人类HLA-A基因的sgRNA、其组合物及应用 |
| WO2026020120A1 (en) * | 2024-07-19 | 2026-01-22 | University Of Massachusetts | Increasing cas9 genome editing fidelity through attenuation of guide rna watson-crick base pairing potential |
Family Cites Families (161)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5132418A (en) | 1980-02-29 | 1992-07-21 | University Patents, Inc. | Process for preparing polynucleotides |
| US4500707A (en) | 1980-02-29 | 1985-02-19 | University Patents, Inc. | Nucleosides useful in the preparation of polynucleotides |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4973679A (en) | 1981-03-27 | 1990-11-27 | University Patents, Inc. | Process for oligonucleo tide synthesis using phosphormidite intermediates |
| US4668777A (en) | 1981-03-27 | 1987-05-26 | University Patents, Inc. | Phosphoramidite nucleoside compounds |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US5153319A (en) | 1986-03-31 | 1992-10-06 | University Patents, Inc. | Process for preparing polynucleotides |
| US5262530A (en) | 1988-12-21 | 1993-11-16 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5047524A (en) | 1988-12-21 | 1991-09-10 | Applied Biosystems, Inc. | Automated system for polynucleotide synthesis and purification |
| US5032401A (en) | 1989-06-15 | 1991-07-16 | Alpha Beta Technology | Glucan drug delivery system and adjuvant |
| US5700642A (en) | 1995-05-22 | 1997-12-23 | Sri International | Oligonucleotide sizing using immobilized cleavable primers |
| US7371580B2 (en) | 2001-08-24 | 2008-05-13 | Agilent Technologies, Inc. | Use of unstructured nucleic acids in assaying nucleic acid molecules |
| US7682828B2 (en) | 2003-11-26 | 2010-03-23 | Whitehead Institute For Biomedical Research | Methods for reprogramming somatic cells |
| WO2005065419A2 (en) | 2003-12-29 | 2005-07-21 | Am Biosolutions | Method of culturing cells |
| US7740861B2 (en) | 2004-06-16 | 2010-06-22 | University Of Massachusetts | Drug delivery product and methods |
| US8278104B2 (en) | 2005-12-13 | 2012-10-02 | Kyoto University | Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2 |
| PL2476689T3 (pl) | 2007-05-10 | 2016-04-29 | Agilent Technologies Inc | Tiowęglowe grupy zabezpieczające do syntezy RNA |
| KR101532442B1 (ko) | 2007-12-10 | 2015-06-29 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 효율적인 핵 초기화 방법 |
| CA2695590C (en) | 2008-06-27 | 2018-01-09 | Kyoto University | Method of efficiently establishing induced pluripotent stem cells |
| US20100076183A1 (en) | 2008-09-22 | 2010-03-25 | Dellinger Douglas J | Protected monomer and method of final deprotection for rna synthesis |
| US8900871B2 (en) | 2009-08-07 | 2014-12-02 | Kyoto University | Method of producing induced pluripotent stem cells using inhibitors of P53 |
| JP5682043B2 (ja) | 2009-09-02 | 2015-03-11 | 国立大学法人京都大学 | 安全な多能性幹細胞の選択方法 |
| US9585920B2 (en) | 2011-02-04 | 2017-03-07 | Katherine Rose Kovarik | Method and system for treating cancer cachexia |
| US9528124B2 (en) | 2013-08-27 | 2016-12-27 | Recombinetics, Inc. | Efficient non-meiotic allele introgression |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| EP2836226B1 (en) | 2012-02-24 | 2017-06-28 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
| WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| EP3747891A3 (en) | 2012-05-21 | 2021-03-24 | Agilent Technologies, Inc. | Compositions and methods for conjugating oligonucleotides |
| ES2728782T3 (es) | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| HK1207111A1 (en) | 2012-08-03 | 2016-01-22 | 加利福尼亚大学董事会 | Methods and compositions for controlling gene expression by rna processing |
| US20140075593A1 (en) | 2012-09-07 | 2014-03-13 | Dow Agrosciences Llc | Fluorescence activated cell sorting (facs) enrichment to generate plants |
| BR112015004995B1 (pt) | 2012-09-07 | 2023-05-02 | Sangamo Biosciences, Inc. | Método para modificação do genoma de uma célula, uso de uma célula, semente ou planta obtida pelo referido método e nuclease de dedo de zinco sítio específica |
| UA118090C2 (uk) | 2012-09-07 | 2018-11-26 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив |
| UA119135C2 (uk) | 2012-09-07 | 2019-05-10 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб отримання трансгенної рослини |
| EP4397759A3 (en) | 2012-10-23 | 2024-10-09 | Toolgen Incorporated | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| US20150315576A1 (en) | 2012-11-01 | 2015-11-05 | Massachusetts Institute Of Technology | Genetic device for the controlled destruction of dna |
| CA2890110C (en) | 2012-11-01 | 2023-05-02 | Factor Bioscience Inc. | Methods and products for expressing proteins in cells |
| WO2014089513A1 (en) | 2012-12-06 | 2014-06-12 | Synthetic Genomics, Inc. | Autonomous replication sequences and episomal dna molecules |
| DK3363902T3 (da) | 2012-12-06 | 2020-01-02 | Sigma Aldrich Co Llc | Crispr-baseret genommodifikation og -regulering |
| CA2892821C (en) | 2012-12-06 | 2022-04-26 | Synthetic Genomics, Inc. | Algal mutants having a locked-in high light acclimated phenotype |
| PT2931898E (pt) | 2012-12-12 | 2016-06-16 | Harvard College | Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais |
| MX380562B (es) * | 2012-12-12 | 2025-03-12 | Broad Inst Inc | Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias. |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| WO2014093688A1 (en) | 2012-12-12 | 2014-06-19 | 1Massachusetts Institute Of Technology | Compositions and methods for functional nucleic acid delivery |
| KR102955556B1 (ko) | 2012-12-12 | 2026-04-22 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물 |
| WO2014093694A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP3064585B1 (en) | 2012-12-12 | 2020-02-05 | The Broad Institute, Inc. | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| IL308158A (en) | 2012-12-17 | 2023-12-01 | Harvard College | Rna-guided human genome engineering |
| WO2014153118A1 (en) | 2013-03-14 | 2014-09-25 | The Board Of Trustees Of The Leland Stanford Junior University | Treatment of diseases and conditions associated with dysregulation of mammalian target of rapamycin complex 1 (mtorc1) |
| WO2014159719A1 (en) | 2013-03-14 | 2014-10-02 | Scrips Health | Methods of isolating nucleic acids |
| ES2749624T3 (es) | 2013-03-14 | 2020-03-23 | Caribou Biosciences Inc | Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| US11332719B2 (en) | 2013-03-15 | 2022-05-17 | The Broad Institute, Inc. | Recombinant virus and preparations thereof |
| JP2016512048A (ja) | 2013-03-15 | 2016-04-25 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | ＣＲＩＳＰＲ／Ｃａｓシステムを使用した植物ゲノム操作 |
| US20140349400A1 (en) | 2013-03-15 | 2014-11-27 | Massachusetts Institute Of Technology | Programmable Modification of DNA |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| CN105408483A (zh) | 2013-03-15 | 2016-03-16 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
| US9957515B2 (en) | 2013-03-15 | 2018-05-01 | Cibus Us Llc | Methods and compositions for targeted gene modification |
| US20140364333A1 (en) | 2013-03-15 | 2014-12-11 | President And Fellows Of Harvard College | Methods for Live Imaging of Cells |
| EP2971006A4 (en) | 2013-03-15 | 2017-02-08 | Transposagen Biopharmaceuticals, Inc. | Reproducible method for testis-mediated genetic modification (tgm) and sperm-mediated genetic modification (sgm) |
| EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| ES2747833T3 (es) | 2013-04-04 | 2020-03-11 | Dartmouth College | Composiciones y procedimientos para la escisión in vivo del ADN proviral del vih-1 |
| EP2986709A4 (en) | 2013-04-16 | 2017-03-15 | University Of Washington Through Its Center For Commercialization | Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering |
| WO2014172470A2 (en) | 2013-04-16 | 2014-10-23 | Whitehead Institute For Biomedical Research | Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| EP2997146A4 (en) | 2013-05-15 | 2017-04-26 | Sangamo BioSciences, Inc. | Methods and compositions for treatment of a genetic condition |
| DK3004349T3 (en) | 2013-05-29 | 2018-06-06 | Cellectis Sa | A method for producing precise DNA cleavage using CAS9 nickase activity |
| US9873907B2 (en) | 2013-05-29 | 2018-01-23 | Agilent Technologies, Inc. | Method for fragmenting genomic DNA using CAS9 |
| JP6608807B2 (ja) | 2013-05-29 | 2019-11-20 | セレクティス | Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法 |
| EP3004339B1 (en) | 2013-05-29 | 2021-07-07 | Cellectis | New compact scaffold of cas9 in the type ii crispr system |
| EP3603679B1 (en) | 2013-06-04 | 2022-08-10 | President and Fellows of Harvard College | Rna-guided transcriptional regulation |
| US20150315252A1 (en) | 2013-06-11 | 2015-11-05 | Clontech Laboratories, Inc. | Protein enriched microvesicles and methods of making and using the same |
| EP3008181B1 (en) | 2013-06-11 | 2019-11-06 | The Regents of The University of California | Methods and compositions for target dna modification |
| WO2014204723A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions |
| JP6738728B2 (ja) | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| SG11201510327TA (en) | 2013-06-17 | 2016-01-28 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
| ES2777217T3 (es) | 2013-06-17 | 2020-08-04 | Broad Inst Inc | Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias |
| WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| JP2016528890A (ja) | 2013-07-09 | 2016-09-23 | 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 | ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用 |
| KR20260047646A (ko) | 2013-07-09 | 2026-04-08 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 멀티플렉스 rna-가이드된 게놈 조작 |
| CN105517579B (zh) | 2013-07-10 | 2019-11-15 | 哈佛大学校长及研究员协会 | 用于RNA向导的基因调节和编辑的正交Cas9蛋白 |
| WO2015006437A1 (en) | 2013-07-10 | 2015-01-15 | Majzoub Joseph A | Mrap2 knockouts |
| PL3019619T3 (pl) * | 2013-07-11 | 2022-01-10 | Modernatx, Inc. | Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania |
| US11306328B2 (en) | 2013-07-26 | 2022-04-19 | President And Fellows Of Harvard College | Genome engineering |
| US20150044772A1 (en) | 2013-08-09 | 2015-02-12 | Sage Labs, Inc. | Crispr/cas system-based novel fusion protein and its applications in genome editing |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| CN106133138A (zh) | 2013-08-22 | 2016-11-16 | 先锋国际良种公司 | 使用引导多核苷酸/cas内切核酸酶系统的基因组修饰及其使用方法 |
| KR20160048212A (ko) | 2013-09-04 | 2016-05-03 | 씨에스아이알 | 유전자 발현을 침묵화시키기 위해 유전자 조절 인자들을 표적으로하는 부위-특이적인 뉴클레아제 단일 세포 분석법 |
| WO2015035034A1 (en) | 2013-09-04 | 2015-03-12 | Mice With Horns, Llc | Materials and methods for correcting recessive mutations in animals |
| BR102014021800B1 (pt) | 2013-09-04 | 2022-03-29 | Dow Agrosciences Llc | Método para detectar a integração específica de sítio de uma sequência de doador de polinucleotídeo dentro de um sítio-alvo genômico |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150071946A1 (en) | 2013-09-06 | 2015-03-12 | The Johns Hopkins University | Tumor-specific retrotransposon insertions |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| ES2681622T3 (es) | 2013-09-18 | 2018-09-14 | Kymab Limited | Métodos, células y organismos |
| WO2015040075A1 (en) | 2013-09-18 | 2015-03-26 | Genome Research Limited | Genomic screening methods using rna-guided endonucleases |
| DE102013111099B4 (de) | 2013-10-08 | 2023-11-30 | Eberhard Karls Universität Tübingen Medizinische Fakultät | Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA |
| US20160251675A1 (en) | 2013-10-08 | 2016-09-01 | International Rice Research Institute | Drought-Resistant Cereal Grasses and Related Materials and Methods |
| US20150098954A1 (en) | 2013-10-08 | 2015-04-09 | Elwha Llc | Compositions and Methods Related to CRISPR Targeting |
| WO2015052231A2 (en) | 2013-10-08 | 2015-04-16 | Technical University Of Denmark | Multiplex editing system |
| WO2015066643A1 (en) | 2013-11-04 | 2015-05-07 | Dow Agrosciences Llc | Optimal soybean loci |
| EP3066110B1 (en) | 2013-11-04 | 2021-12-29 | Corteva Agriscience LLC | Optimal maize loci |
| JP6633532B2 (ja) | 2013-11-04 | 2020-01-22 | ダウ アグロサイエンシィズ エルエルシー | 最適なトウモロコシ遺伝子座 |
| BR102014027448A8 (pt) | 2013-11-04 | 2021-08-24 | Dow Agrosciences Llc | cassete de doador de polinucleotídeo, uso de uma planta, de uma parte de planta, ou de uma célula vegetal compreendendo o referido cassete, e método para integração direcionada do mesmo |
| CN105980395A (zh) | 2013-11-04 | 2016-09-28 | 美国陶氏益农公司 | 最优大豆座位 |
| US11326209B2 (en) | 2013-11-07 | 2022-05-10 | Massachusetts Institute Of Technology | Cell-based genomic recorded accumulative memory |
| CN111218447B (zh) | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| WO2015075056A1 (en) | 2013-11-19 | 2015-05-28 | Thermo Fisher Scientific Baltics Uab | Programmable enzymes for isolation of specific dna fragments |
| US9074199B1 (en) | 2013-11-19 | 2015-07-07 | President And Fellows Of Harvard College | Mutant Cas9 proteins |
| US10787684B2 (en) | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
| CN103614415A (zh) * | 2013-11-27 | 2014-03-05 | 苏州同善生物科技有限公司 | 一种基于crispr基因敲除技术建立肥胖症大鼠动物模型的方法 |
| CN105899658B (zh) | 2013-12-12 | 2020-02-18 | 布罗德研究所有限公司 | 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用 |
| EP3835419A1 (en) | 2013-12-12 | 2021-06-16 | The Regents of The University of California | Methods and compositions for modifying a single stranded target nucleic acid |
| WO2015089419A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| KR20160097338A (ko) | 2013-12-12 | 2016-08-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도 |
| CN111206032B (zh) * | 2013-12-12 | 2024-07-19 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
| JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物 |
| CA2932439A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute, Inc. | Crispr-cas systems and methods for altering expression of gene products, structural information and inducible modular cas enzymes |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| US9938521B2 (en) * | 2014-03-10 | 2018-04-10 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10) |
| EP3981876A1 (en) | 2014-03-26 | 2022-04-13 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| US20150344836A1 (en) | 2014-05-30 | 2015-12-03 | Ohio State Innovation Foundation | Agrobacterium Strains for Plant Transformation and Related Materials and Methods |
| WO2015184262A1 (en) | 2014-05-30 | 2015-12-03 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods of delivering treatments for latent viral infections |
| WO2015191693A2 (en) * | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| AU2015280069B2 (en) | 2014-06-23 | 2021-08-12 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
| US9738897B2 (en) | 2014-06-23 | 2017-08-22 | Regeneron Pharmaceuticals, Inc. | Nuclease-mediated DNA assembly |
| WO2015200725A1 (en) | 2014-06-25 | 2015-12-30 | Cold Spring Harbor Laboratory | Methods and compositions for inhibiting growth and epithelial to mesenchymal transition (emt) in cancer cells |
| WO2015200555A2 (en) | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
| US9616090B2 (en) | 2014-07-30 | 2017-04-11 | Sangamo Biosciences, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| ES2730378T3 (es) | 2014-08-27 | 2019-11-11 | Caribou Biosciences Inc | Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9 |
| JP6594955B2 (ja) | 2014-08-27 | 2019-10-23 | ニユー・イングランド・バイオレイブス・インコーポレイテツド | シントン形成 |
| WO2016049163A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder |
| WO2016049251A1 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes |
| WO2016049024A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo |
| RU2721799C2 (ru) | 2014-09-26 | 2020-05-22 | Филип Моррис Продактс С.А. | Уменьшение содержания табак-специфичных нитрозаминов посредством изменения пути ассимиляции нитратов |
| EP3198036A4 (en) | 2014-09-26 | 2018-08-08 | Two Pore Guys, Inc. | Target sequence detection by nanopore sensing of synthetic probes |
| WO2016073079A2 (en) | 2014-09-26 | 2016-05-12 | Yale University | Compositions and methods for biocontainment of microorganisms |
| EP3197270B1 (en) | 2014-09-26 | 2025-12-03 | Pioneer Hi-Bred International, Inc. | Wheat ms1 polynucleotides, polypeptides, and methods of use |
| CN113930455A (zh) | 2014-10-09 | 2022-01-14 | 生命技术公司 | Crispr寡核苷酸和基因剪辑 |
| AU2015330855A1 (en) | 2014-10-09 | 2017-04-27 | Celularity Inc. | Placenta-derived adherent cell exosomes and uses thereof |
| TWI716367B (zh) | 2014-10-31 | 2021-01-21 | 麻省理工學院 | 用於常間回文重複序列叢集（ｃｒｉｓｐｒ）之大量平行組合性基因學 |
| US9816080B2 (en) | 2014-10-31 | 2017-11-14 | President And Fellows Of Harvard College | Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs) |
| KR102546296B1 (ko) | 2014-10-31 | 2023-06-21 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 변형된 t 세포에서 유전자 발현의 변경 및 그의 용도 |
| CA2969619A1 (en) | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| JP2017538427A (ja) * | 2014-12-18 | 2017-12-28 | インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド | Ｃｒｉｓｐｒ系組成物及び使用方法 |
| CN107787367B (zh) | 2015-04-06 | 2021-10-26 | 里兰斯坦福初级大学理事会 | 用于crispr/cas介导的基因调控的化学修饰的引导rna |
| 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 |
| EP3390624A4 (en) | 2015-12-18 | 2019-07-10 | The Regents of The University of California | Modified site-directed modifying polypeptides and methods of use thereof |
| WO2017106657A1 (en) | 2015-12-18 | 2017-06-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| EP3390632B1 (en) | 2015-12-18 | 2025-09-10 | Danisco US Inc. | Methods and compositions for polymerase ii (pol-ii) based guide rna expression |
| EP3392337B1 (en) | 2015-12-18 | 2024-03-06 | Japan Science and Technology Agency | Genetic modification non-human organism, egg cells, fertilized eggs, and method for modifying target genes |
| WO2017106767A1 (en) | 2015-12-18 | 2017-06-22 | The Scripps Research Institute | Production of unnatural nucleotides using a crispr/cas9 system |
| EP3708665A1 (en) | 2015-12-18 | 2020-09-16 | Danisco US Inc. | Methods and compositions for t-rna based guide rna expression |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
-
2016
- 2016-04-05 CN CN201680032647.XA patent/CN107787367B/zh active Active
- 2016-04-05 KR KR1020177031965A patent/KR102648489B1/ko active Active
- 2016-04-05 WO PCT/US2016/026028 patent/WO2016164356A1/en not_active Ceased
- 2016-04-05 CN CN202111196588.9A patent/CN114231527B/zh active Active
- 2016-04-05 ES ES16720220T patent/ES2884838T3/es active Active
- 2016-04-05 CA CA2981715A patent/CA2981715A1/en active Pending
- 2016-04-05 KR KR1020247008357A patent/KR102888521B1/ko active Active
- 2016-04-05 AU AU2016246450A patent/AU2016246450B2/en active Active
- 2016-04-05 JP JP2017552447A patent/JP6873911B2/ja active Active
- 2016-04-05 EP EP16720220.9A patent/EP3280803B1/en active Active
-
2017
- 2017-10-03 US US15/724,073 patent/US11306309B2/en active Active
-
2022
- 2022-03-14 US US17/694,336 patent/US20220195426A1/en not_active Abandoned
- 2022-03-14 US US17/694,361 patent/US11851652B2/en active Active
- 2022-03-14 US US17/694,310 patent/US11535846B2/en active Active
- 2022-06-17 AU AU2022204254A patent/AU2022204254B2/en active Active
-
2024
- 2024-01-09 US US18/408,504 patent/US20240401034A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7698828B2 (ja) | Ｒｎａを編集する方法および組成物 | |
| US11332758B2 (en) | Methods and products for expressing proteins in cells | |
| US20230365962A1 (en) | Targeted rna editing by leveraging endogenous adar using engineered rnas | |
| US12622931B2 (en) | Compositions and methods for treating alpha-1 antitrypsin deficiency | |
| ES2985812T3 (es) | Edición genómica de precisión de alto rendimiento | |
| US11492670B2 (en) | Compositions and methods for targeting cancer-specific sequence variations | |
| JP2022009734A (ja) | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 | |
| JP6997623B2 (ja) | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 | |
| TW202028466A (zh) | 編輯RNAs的方法以及組成物 | |
| JP2021511038A5 (enExample) | ||
| JP2023523237A (ja) | ｓｎＲＮＡ構成要素を使用する組成物及び方法 | |
| JP2019524149A5 (enExample) | ||
| JP2022549138A5 (enExample) | ||
| JP2016537028A5 (enExample) | ||
| US20240309368A1 (en) | Targeted rna editing by leveraging endogenous adar using engineered rnas | |
| CA2968336A1 (en) | Construct for site directed editing of an adenosine nucleotide in target rna | |
| AU2022353130A1 (en) | Compositions and methods for treating hepatitis b virus infection | |
| Park et al. | Applications of CRISPR technologies to the development of gene and cell therapy | |
| WO2023046086A1 (zh) | 一种碱基编辑系统及其应用 | |
| CN117980482A (zh) | Rbm20突变的基因组编辑 | |
| WO2021182517A1 (ja) | －１フレームシフトを誘導するための１本鎖核酸分子及び組成物 | |
| Reza et al. | Therapeutic genome mutagenesis using synthetic donor DNA and triplex-forming molecules | |
| US20240325454A1 (en) | Genomic editing of rbm20 mutations | |
| Liu | The use of new CRISPR tools in cardiovascular research and medicine | |
| CN120624447A (zh) | 一种靶向前激肽释放酶的gRNA、重组载体、RNA递送系统、基因编辑系统、重组细胞及其应用 |