JP2019524149A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019524149A5 JP2019524149A5 JP2019510339A JP2019510339A JP2019524149A5 JP 2019524149 A5 JP2019524149 A5 JP 2019524149A5 JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019510339 A JP2019510339 A JP 2019510339A JP 2019524149 A5 JP2019524149 A5 JP 2019524149A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- seq
- crispr
- cas9 system
- snp
- 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
- 108091033409 CRISPR Proteins 0.000 claims 125
- 238000010354 CRISPR gene editing Methods 0.000 claims 56
- 239000002773 nucleotide Substances 0.000 claims 32
- 125000003729 nucleotide group Chemical group 0.000 claims 32
- 230000035772 mutation Effects 0.000 claims 28
- 206010011005 corneal dystrophy Diseases 0.000 claims 26
- 208000003923 Hereditary Corneal Dystrophies Diseases 0.000 claims 20
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 18
- 101710163270 Nuclease Proteins 0.000 claims 17
- 206010064571 Gene mutation Diseases 0.000 claims 10
- 210000000130 stem cell Anatomy 0.000 claims 10
- 108700006666 betaIG-H3 Proteins 0.000 claims 9
- 108020004414 DNA Proteins 0.000 claims 6
- 201000004889 corneal granular dystrophy Diseases 0.000 claims 6
- 208000014706 granular corneal dystrophy Diseases 0.000 claims 6
- 230000001717 pathogenic effect Effects 0.000 claims 6
- 102000053602 DNA Human genes 0.000 claims 5
- 210000004027 cell Anatomy 0.000 claims 5
- 102000039446 nucleic acids Human genes 0.000 claims 5
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 102200040436 rs121909211 Human genes 0.000 claims 5
- 241000191940 Staphylococcus Species 0.000 claims 4
- 241000194017 Streptococcus Species 0.000 claims 4
- 101150040304 TGFBI gene Proteins 0.000 claims 4
- 102220601842 Transforming growth factor-beta-induced protein ig-h3_N622K_mutation Human genes 0.000 claims 4
- 210000004087 cornea Anatomy 0.000 claims 4
- 102200040405 rs121909211 Human genes 0.000 claims 4
- 102200040244 rs121909215 Human genes 0.000 claims 4
- 102200040225 rs121909217 Human genes 0.000 claims 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 3
- 102220600069 Transforming growth factor-beta-induced protein ig-h3_L527R_mutation Human genes 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102200040258 rs121909208 Human genes 0.000 claims 3
- 102200040253 rs121909209 Human genes 0.000 claims 3
- 102200040434 rs121909210 Human genes 0.000 claims 3
- 201000004173 Epithelial basement membrane dystrophy Diseases 0.000 claims 2
- 102100028953 Gelsolin Human genes 0.000 claims 2
- 101001059150 Homo sapiens Gelsolin Proteins 0.000 claims 2
- 101000975472 Homo sapiens Keratin, type I cytoskeletal 12 Proteins 0.000 claims 2
- 101001056469 Homo sapiens Keratin, type II cytoskeletal 3 Proteins 0.000 claims 2
- 101000608653 Homo sapiens UbiA prenyltransferase domain-containing protein 1 Proteins 0.000 claims 2
- 102100023967 Keratin, type I cytoskeletal 12 Human genes 0.000 claims 2
- 102100025759 Keratin, type II cytoskeletal 3 Human genes 0.000 claims 2
- 102220620636 Lysosomal acid glucosylceramidase_L509P_mutation Human genes 0.000 claims 2
- 208000009869 Neu-Laxova syndrome Diseases 0.000 claims 2
- 108010077850 Nuclear Localization Signals Proteins 0.000 claims 2
- 102220552750 Proprotein convertase subtilisin/kexin type 9_F515L_mutation Human genes 0.000 claims 2
- 102220569565 Ribonuclease P protein subunit p14_L558P_mutation Human genes 0.000 claims 2
- 102220600031 Transforming growth factor-beta-induced protein ig-h3_A546T_mutation Human genes 0.000 claims 2
- 102220601844 Transforming growth factor-beta-induced protein ig-h3_G594V_mutation Human genes 0.000 claims 2
- 102220601850 Transforming growth factor-beta-induced protein ig-h3_H572R_mutation Human genes 0.000 claims 2
- 102220601839 Transforming growth factor-beta-induced protein ig-h3_H626P_mutation Human genes 0.000 claims 2
- 102220601833 Transforming growth factor-beta-induced protein ig-h3_H626R_mutation Human genes 0.000 claims 2
- 102220600360 Transforming growth factor-beta-induced protein ig-h3_L518P_mutation Human genes 0.000 claims 2
- 102220600362 Transforming growth factor-beta-induced protein ig-h3_L518R_mutation Human genes 0.000 claims 2
- 102220601849 Transforming growth factor-beta-induced protein ig-h3_L569R_mutation Human genes 0.000 claims 2
- 102220601846 Transforming growth factor-beta-induced protein ig-h3_N622H_mutation Human genes 0.000 claims 2
- 102220600049 Transforming growth factor-beta-induced protein ig-h3_T538R_mutation Human genes 0.000 claims 2
- 102220600365 Transforming growth factor-beta-induced protein ig-h3_V505D_mutation Human genes 0.000 claims 2
- 102220600051 Transforming growth factor-beta-induced protein ig-h3_V539D_mutation Human genes 0.000 claims 2
- 102220601789 Transforming growth factor-beta-induced protein ig-h3_V631D_mutation Human genes 0.000 claims 2
- 102100039547 UbiA prenyltransferase domain-containing protein 1 Human genes 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 102000054765 polymorphisms of proteins Human genes 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- 230000001105 regulatory effect Effects 0.000 claims 2
- 102200040404 rs121909210 Human genes 0.000 claims 2
- 102200040401 rs121909212 Human genes 0.000 claims 2
- 102200040397 rs121909214 Human genes 0.000 claims 2
- 102200040403 rs121909216 Human genes 0.000 claims 2
- 102200137143 rs121918254 Human genes 0.000 claims 2
- 102200040392 rs267607109 Human genes 0.000 claims 2
- 102200040393 rs267607110 Human genes 0.000 claims 2
- 102220055023 rs369100046 Human genes 0.000 claims 2
- 102200055968 rs374603772 Human genes 0.000 claims 2
- 102200004603 rs58343600 Human genes 0.000 claims 2
- 102220218946 rs762018538 Human genes 0.000 claims 2
- 102220240577 rs764854618 Human genes 0.000 claims 2
- 102220323268 rs772652517 Human genes 0.000 claims 2
- 102220392615 rs774013935 Human genes 0.000 claims 2
- 102200040399 rs777288957 Human genes 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 102220531909 Gelsolin_D214Y_mutation Human genes 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 101000894525 Homo sapiens Transforming growth factor-beta-induced protein ig-h3 Proteins 0.000 claims 1
- 102220637260 Keratin, type I cytoskeletal 12_L132V_mutation Human genes 0.000 claims 1
- 102000008300 Mutant Proteins Human genes 0.000 claims 1
- 108010021466 Mutant Proteins Proteins 0.000 claims 1
- 102100021398 Transforming growth factor-beta-induced protein ig-h3 Human genes 0.000 claims 1
- 102220600114 Transforming growth factor-beta-induced protein ig-h3_D123H_mutation Human genes 0.000 claims 1
- 102220600131 Transforming growth factor-beta-induced protein ig-h3_V113I_mutation Human genes 0.000 claims 1
- 102220540617 UbiA prenyltransferase domain-containing protein 1_A97T_mutation Human genes 0.000 claims 1
- 102220540625 UbiA prenyltransferase domain-containing protein 1_D112N_mutation Human genes 0.000 claims 1
- 102220540610 UbiA prenyltransferase domain-containing protein 1_D118G_mutation Human genes 0.000 claims 1
- 102220540431 UbiA prenyltransferase domain-containing protein 1_D240N_mutation Human genes 0.000 claims 1
- 102220540626 UbiA prenyltransferase domain-containing protein 1_G98S_mutation Human genes 0.000 claims 1
- 102220540606 UbiA prenyltransferase domain-containing protein 1_L121F_mutation Human genes 0.000 claims 1
- 102220540600 UbiA prenyltransferase domain-containing protein 1_L188H_mutation Human genes 0.000 claims 1
- 102220540595 UbiA prenyltransferase domain-containing protein 1_V122E_mutation Human genes 0.000 claims 1
- 102220540602 UbiA prenyltransferase domain-containing protein 1_V122G_mutation Human genes 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 210000003527 eukaryotic cell Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 102200059610 rs118203945 Human genes 0.000 claims 1
- 102200059669 rs118203946 Human genes 0.000 claims 1
- 102200059615 rs118203947 Human genes 0.000 claims 1
- 102200059663 rs118203948 Human genes 0.000 claims 1
- 102200059672 rs118203949 Human genes 0.000 claims 1
- 102200059611 rs118203950 Human genes 0.000 claims 1
- 102200059492 rs118203951 Human genes 0.000 claims 1
- 102200059666 rs118203952 Human genes 0.000 claims 1
- 102200059673 rs118203953 Human genes 0.000 claims 1
- 102220086650 rs142371511 Human genes 0.000 claims 1
- 102200081932 rs2274064 Human genes 0.000 claims 1
- 102200004717 rs267607386 Human genes 0.000 claims 1
- 102200004568 rs267607387 Human genes 0.000 claims 1
- 102220024596 rs267607431 Human genes 0.000 claims 1
- 102200004567 rs28936695 Human genes 0.000 claims 1
- 102200004548 rs57218384 Human genes 0.000 claims 1
- 102200004597 rs57218384 Human genes 0.000 claims 1
- 102200053908 rs57872071 Human genes 0.000 claims 1
- 102200004598 rs58038639 Human genes 0.000 claims 1
- 102200004576 rs58162394 Human genes 0.000 claims 1
- 102200004553 rs58410481 Human genes 0.000 claims 1
- 102200004569 rs58864803 Human genes 0.000 claims 1
- 102200004601 rs58918655 Human genes 0.000 claims 1
- 102200004582 rs59202432 Human genes 0.000 claims 1
- 102200004607 rs59350319 Human genes 0.000 claims 1
- 102220024224 rs59465138 Human genes 0.000 claims 1
- 102200053910 rs60410063 Human genes 0.000 claims 1
- 102220024225 rs61167390 Human genes 0.000 claims 1
- 102200004596 rs61282718 Human genes 0.000 claims 1
- 102200004578 rs62635290 Human genes 0.000 claims 1
- 102220118475 rs781152868 Human genes 0.000 claims 1
- 102200004551 rs886038212 Human genes 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021214009A JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662377586P | 2016-08-20 | 2016-08-20 | |
| US62/377,586 | 2016-08-20 | ||
| US201762462808P | 2017-02-23 | 2017-02-23 | |
| US62/462,808 | 2017-02-23 | ||
| US201762501750P | 2017-05-05 | 2017-05-05 | |
| US62/501,750 | 2017-05-05 | ||
| PCT/US2017/047861 WO2018039145A1 (en) | 2016-08-20 | 2017-08-21 | Single guide rna, crispr/cas9 systems, and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021214009A Division JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019524149A JP2019524149A (ja) | 2019-09-05 |
| JP2019524149A5 true JP2019524149A5 (enExample) | 2020-10-01 |
Family
ID=61246315
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019510339A Pending JP2019524149A (ja) | 2016-08-20 | 2017-08-21 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2021214009A Pending JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A Pending JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021214009A Pending JP2022046694A (ja) | 2016-08-20 | 2021-12-28 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
| JP2024002942A Pending JP2024041905A (ja) | 2016-08-20 | 2024-01-12 | 一本鎖ガイドＲＮＡ、ＣＲＩＳＰＲ／Ｃａｓ９システム、及びそれらの使用方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20190185850A1 (enExample) |
| EP (1) | EP3500677A4 (enExample) |
| JP (3) | JP2019524149A (enExample) |
| KR (2) | KR102594051B1 (enExample) |
| CN (1) | CN109963945A (enExample) |
| WO (1) | WO2018039145A1 (enExample) |
Families Citing this family (56)
| 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 |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| 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 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| WO2017083852A1 (en) | 2015-11-13 | 2017-05-18 | MOORE, Tara | Methods for the treatment of corneal dystrophies |
| EA201891317A3 (ru) | 2015-11-30 | 2019-04-30 | Дьюк Юниверсити | Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения |
| EP3443081A4 (en) | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| US12214056B2 (en) | 2016-07-19 | 2025-02-04 | Duke University | Therapeutic applications of CPF1-based genome editing |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| KR102594051B1 (ko) * | 2016-08-20 | 2023-10-26 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
| WO2020046861A1 (en) * | 2018-08-27 | 2020-03-05 | Avellino Lab Usa, Inc. | Crispr/cas9 systems, and methods of use thereof |
| WO2019165322A1 (en) * | 2018-02-22 | 2019-08-29 | Moore Tara | Crispr/cas9 systems, and methods of use thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| 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 |
| 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 |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| 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 |
| CN111065736A (zh) * | 2017-06-07 | 2020-04-24 | 国立大学法人东京大学 | 针对颗粒状角膜变性症的基因治疗药物 |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019183150A1 (en) * | 2018-03-19 | 2019-09-26 | Casebia Therapeutics Limited Liability Partnership | Novel rna-programmable endonuclease systems and uses thereof |
| WO2019215216A1 (en) * | 2018-05-08 | 2019-11-14 | Katholieke Universiteit Leuven | Biosensor |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CA3121191A1 (en) | 2018-11-28 | 2020-06-04 | Crispr Therapeutics Ag | Optimized mrna encoding cas9 for use in lnps |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN110551763B (zh) * | 2019-08-08 | 2023-03-10 | 复旦大学 | CRISPR/SlutCas9基因编辑系统及其应用 |
| CN110499335B (zh) * | 2019-08-08 | 2023-03-28 | 复旦大学 | CRISPR/SauriCas9基因编辑系统及其应用 |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| CN115515968A (zh) * | 2019-12-11 | 2022-12-23 | 阿维利诺美国实验室股份有限公司 | 使用短干扰RNA对具有R124H突变的转化生长因子β诱导基因的等位基因特异性沉默 |
| GB2614813B (en) | 2020-05-08 | 2025-05-07 | Harvard College | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| CN111926015B (zh) * | 2020-08-24 | 2022-07-15 | 武汉纽福斯生物科技有限公司 | 寡核苷酸、病毒载体及其应用和RNAi药物制剂 |
| WO2022170059A1 (en) * | 2021-02-05 | 2022-08-11 | Christiana Care Health Services, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| WO2023034704A1 (en) * | 2021-08-31 | 2023-03-09 | The Regents Of The University Of California | Products and methods for annotating gene function using locally haploid, human non-cancer cells |
| WO2023147428A2 (en) * | 2022-01-26 | 2023-08-03 | Orthobio Therapeutics, Inc. | Gene editing to improve joint function |
| EP4463555A1 (en) * | 2022-02-25 | 2024-11-20 | Incisive Genetics, Inc. | Gene editing reporter system and guide rna and composition related thereto; composition and method for knocking out dna with more than two grnas; gene editing in the eye; and gene editing using base editors |
| CN115948537B (zh) * | 2022-12-19 | 2024-04-09 | 湖南家辉生物技术有限公司 | 一种基因chst3复合杂合突变的应用及检测试剂和应用 |
| CN116286823A (zh) * | 2023-04-04 | 2023-06-23 | 尧唐(上海)生物科技有限公司 | 靶向TGFBI基因的sgRNA、载体及其应用 |
| WO2025043140A1 (en) * | 2023-08-23 | 2025-02-27 | Emendobio Inc. | Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele |
| CN119464347B (zh) * | 2024-11-26 | 2025-10-03 | 扬州大学 | 基于CRISPR-Cas9系统的噬菌体基因组编辑载体及其编辑方法和应用 |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
| US9895451B2 (en) | 2011-12-02 | 2018-02-20 | Yale University | Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof |
| WO2013176772A1 (en) * | 2012-05-25 | 2013-11-28 | The Regents Of The University Of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| RS60838B1 (sr) | 2012-08-29 | 2020-10-30 | Sangamo Therapeutics Inc | Postupci i kompozicije za tretman genskog stanja |
| ES2713243T3 (es) * | 2012-12-06 | 2019-05-20 | Sigma Aldrich Co Llc | Modificación y regulación del genoma basada en CRISPR |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| 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 |
| AU2013359199C1 (en) | 2012-12-12 | 2021-06-17 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| EP4234696A3 (en) | 2012-12-12 | 2023-09-06 | The Broad Institute Inc. | Crispr-cas component systems, methods and compositions for sequence manipulation |
| DK2921557T3 (en) | 2012-12-12 | 2016-11-07 | Broad Inst Inc | Design of systems, methods and optimized sequence manipulation guide compositions |
| IL307735A (en) | 2012-12-12 | 2023-12-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| PL2931898T3 (pl) | 2012-12-12 | 2016-09-30 | Le Cong | Projektowanie i optymalizacja systemów, sposoby i kompozycje do manipulacji sekwencją z domenami funkcjonalnymi |
| EP2975117B1 (en) * | 2013-03-11 | 2018-01-10 | JCR Pharmaceuticals Co., Ltd. | Method for producing human corneal epithelium sheet |
| WO2014186585A2 (en) | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| AU2014281026B2 (en) * | 2013-06-17 | 2020-05-28 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| IL303973A (en) * | 2013-07-09 | 2023-08-01 | Harvard College | Multiplexed RNA-guided 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 |
| US10787684B2 (en) * | 2013-11-19 | 2020-09-29 | President And Fellows Of Harvard College | Large gene excision and insertion |
| 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 |
| AU2016335572B2 (en) * | 2015-10-09 | 2022-12-08 | The Children's Hospital Of Philadelphia | Compositions and methods for treating Huntington's disease and related disorders |
| WO2017083852A1 (en) * | 2015-11-13 | 2017-05-18 | MOORE, Tara | Methods for the treatment of corneal dystrophies |
| KR102594051B1 (ko) * | 2016-08-20 | 2023-10-26 | 아벨리노 랩 유에스에이, 인크. | 단일 가이드 RNA, CRISPR/Cas9 시스템, 및 이의 사용방법 |
-
2017
- 2017-08-21 KR KR1020197007806A patent/KR102594051B1/ko active Active
- 2017-08-21 EP EP17844228.1A patent/EP3500677A4/en not_active Withdrawn
- 2017-08-21 KR KR1020237036111A patent/KR20230155013A/ko not_active Ceased
- 2017-08-21 WO PCT/US2017/047861 patent/WO2018039145A1/en not_active Ceased
- 2017-08-21 CN CN201780064151.5A patent/CN109963945A/zh active Pending
- 2017-08-21 US US16/326,908 patent/US20190185850A1/en not_active Abandoned
- 2017-08-21 JP JP2019510339A patent/JP2019524149A/ja active Pending
-
2021
- 2021-12-28 JP JP2021214009A patent/JP2022046694A/ja active Pending
-
2024
- 2024-01-12 JP JP2024002942A patent/JP2024041905A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019524149A5 (enExample) | ||
| US20230407341A1 (en) | Using Truncated Guide RNAs (tru-gRNAs) to Increase Specificity for RNA-Guided Genome Editing | |
| JP7379447B2 (ja) | ゲノム編集分子の細胞内送達のためのペプチドおよびナノ粒子 | |
| CN107787367B (zh) | 用于crispr/cas介导的基因调控的化学修饰的引导rna | |
| CN108350454B (zh) | 等位基因选择性基因编辑及其用途 | |
| JP2023113627A (ja) | シトシンからグアニンへの塩基編集因子 | |
| JP2016537028A5 (enExample) | ||
| WO2015115903A1 (en) | Site-specific dna break-induced genome editing using engineered nucleases | |
| FI3802807T3 (fi) | Rna-ohjattuja nukleaaseja ja niiden aktiivisia fragmentteja ja variantteja ja menetelmiä niiden käyttämiseksi | |
| JP2019522461A5 (enExample) | ||
| JP2018527013A5 (enExample) | ||
| JP2019115348A5 (enExample) | ||
| JP2018518182A5 (enExample) | ||
| KR20190090848A (ko) | 변형된 가이드 rna | |
| JP2018534950A5 (enExample) | ||
| IL302205B2 (en) | Rna-guided transcriptional regulation | |
| JP2016501532A5 (enExample) | ||
| JP2022023144A5 (enExample) | ||
| RU2020100919A (ru) | СПОСОБ НОКАУТИРОВАНИЯ ГЕНА-МИШЕНИ В T-КЛЕТКЕ IN VITRO И crRNA, ПРИМЕНЯЕМАЯ В ДАННОМ СПОСОБЕ | |
| CN109789185A (zh) | 用于治疗眼部疾病的含cas9蛋白和向导rna的药物组合物 | |
| ES2285118T5 (es) | Un método para el desarrollo molecular in vitro de una función proteica. | |
| WO2023220742A3 (en) | Abca4 trans-splicing molecules | |
| Chen et al. | Mitochondria isolated from liver contain the essential factors required for RNA/DNA oligonucleotide-targeted gene repair | |
| Eslahi et al. | New advancements in CRISPR based gene therapy of Duchenne muscular dystrophy | |
| JPWO2020223514A5 (enExample) |