JP2018516978A5 - - Google Patents
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- JP2018516978A5 JP2018516978A5 JP2018511592A JP2018511592A JP2018516978A5 JP 2018516978 A5 JP2018516978 A5 JP 2018516978A5 JP 2018511592 A JP2018511592 A JP 2018511592A JP 2018511592 A JP2018511592 A JP 2018511592A JP 2018516978 A5 JP2018516978 A5 JP 2018516978A5
- Authority
- JP
- Japan
- Prior art keywords
- guide rna
- cas
- cas protein
- mutation
- promoter
- 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
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021189812A JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A JP2024073536A (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562162720P | 2015-05-16 | 2015-05-16 | |
| US62/162,720 | 2015-05-16 | ||
| PCT/US2016/027987 WO2016186772A2 (en) | 2015-05-16 | 2016-04-15 | Gene editing of deep intronic mutations |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021189812A Division JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018516978A JP2018516978A (ja) | 2018-06-28 |
| JP2018516978A5 true JP2018516978A5 (enExample) | 2019-05-23 |
| JP6985250B2 JP6985250B2 (ja) | 2021-12-22 |
Family
ID=55809259
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018511592A Active JP6985250B2 (ja) | 2015-05-16 | 2016-04-15 | 深部イントロン突然変異の遺伝子編集 |
| JP2021189812A Pending JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A Pending JP2024073536A (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021189812A Pending JP2022031769A (ja) | 2015-05-16 | 2021-11-24 | 深部イントロン突然変異の遺伝子編集 |
| JP2024038537A Pending JP2024073536A (ja) | 2015-05-16 | 2024-03-13 | 深部イントロン突然変異の遺伝子編集 |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US11896651B2 (enExample) |
| EP (1) | EP3298134B8 (enExample) |
| JP (3) | JP6985250B2 (enExample) |
| KR (2) | KR20230142649A (enExample) |
| CN (3) | CN114657180A (enExample) |
| AU (2) | AU2016265255B2 (enExample) |
| BR (1) | BR112017024514A2 (enExample) |
| CA (1) | CA2986021A1 (enExample) |
| ES (1) | ES2952598T3 (enExample) |
| IL (2) | IL296365A (enExample) |
| MX (2) | MX2017014665A (enExample) |
| PL (1) | PL3298134T3 (enExample) |
| PT (1) | PT3298134T (enExample) |
| RU (2) | RU2759335C2 (enExample) |
| SG (2) | SG10201912907PA (enExample) |
| WO (1) | WO2016186772A2 (enExample) |
Families Citing this family (76)
| 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 |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP3957735A1 (en) | 2014-03-05 | 2022-02-23 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| DK3116997T3 (da) | 2014-03-10 | 2019-08-19 | Editas Medicine Inc | Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10) |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| US11242525B2 (en) | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| EP3540061A1 (en) | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| BR112017010547A2 (pt) | 2014-11-21 | 2018-02-27 | Regeneron Pharma | métodos para produção de uma modificação bialélica, modificação de um genoma dentro de uma célula, produção de uma geração f0 de um animal não humano e identificação de uma inserção de um inserto de ácido nucleico. |
| KR102535217B1 (ko) | 2015-04-24 | 2023-05-19 | 에디타스 메디신, 인코포레이티드 | Ｃａｓ9 분자/가이드 ｒｎａ 분자 복합체의 평가 |
| WO2016186772A2 (en) * | 2015-05-16 | 2016-11-24 | Genzyme Corporation | Gene editing of deep intronic mutations |
| EP4115894A1 (en) * | 2015-06-19 | 2023-01-11 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| IL294014B2 (en) | 2015-10-23 | 2024-07-01 | Harvard College | Nucleobase editors and uses thereof |
| US11512311B2 (en) | 2016-03-25 | 2022-11-29 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency |
| IL262888B1 (en) | 2016-05-20 | 2025-10-01 | Regeneron Pharma | Methods for breaking immunological tolerance using multiple guide RNAs |
| CA3032822A1 (en) | 2016-08-02 | 2018-02-08 | Editas Medicine, Inc. | Compositions and methods for treating cep290 associated disease |
| 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 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| IT201600102542A1 (it) * | 2016-10-12 | 2018-04-12 | Univ Degli Studi Di Trento | Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza. |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| CA3046076A1 (en) | 2016-12-05 | 2018-06-14 | Editas Medicine, Inc. | Systems and methods for one-shot guide rna (ogrna) targeting of endogenous and source dna |
| 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 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| GB2575930A (en) | 2017-03-23 | 2020-01-29 | Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| EP3622073A4 (en) * | 2017-05-09 | 2021-01-06 | University of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| EP3622070A2 (en) | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| 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 |
| 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 |
| EP3684937A4 (en) | 2017-09-22 | 2021-06-02 | University of Massachusetts | NEW DUAL SOD1 EXPRESSION VECTORS AND RELATED USES |
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| JP7522656B2 (ja) | 2017-11-21 | 2024-07-25 | クリスパー セラピューティクス アーゲー | 常染色体優性網膜色素変性の処置のための材料および方法 |
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| EP3765614A1 (en) | 2018-03-14 | 2021-01-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| US20210017509A1 (en) * | 2018-03-23 | 2021-01-21 | The Trustees Of Columbia University In The City Of New York | Gene Editing for Autosomal Dominant Diseases |
| CA3094828A1 (en) * | 2018-03-23 | 2019-09-26 | Massachusetts Eye And Ear Infirmary | Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome |
| US12121563B2 (en) | 2018-04-09 | 2024-10-22 | Allen Institute | Rescuing voltage-gated sodium channel function in inhibitory neurons |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| EP3833680A4 (en) * | 2018-05-30 | 2022-05-25 | The Regents of the University of California | GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED HYPER-IGM SYNDROME (XHIM) |
| GB201809588D0 (en) * | 2018-06-12 | 2018-07-25 | Univ Bristol | Materials and methods for modulating intraocular and intracranial pressure |
| CA3108376A1 (en) * | 2018-08-02 | 2020-02-06 | Editas Medicine, Inc. | Compositions and methods for treating cep290-associated disease |
| KR102158428B1 (ko) * | 2018-08-16 | 2020-09-22 | (주)라트바이오 | 인위적 뉴클레아제를 생산하는 형질전환 배아 및 형질전환 동물의 제작 방법 |
| US12472268B2 (en) | 2018-10-01 | 2025-11-18 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| JP7695192B2 (ja) | 2018-10-08 | 2025-06-18 | アレン インスティテュート | 介在ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| JP7704998B2 (ja) * | 2018-11-21 | 2025-07-09 | 武田薬品工業株式会社 | 組換えウイルスベクター及びそれの産生のための核酸 |
| IT201800020230A1 (it) * | 2018-12-19 | 2020-06-19 | Univ Degli Studi Di Siena | Sistema CRISPR-Cas per la terapia genica. |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020176747A1 (en) * | 2019-02-28 | 2020-09-03 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| DE112020001342T5 (de) | 2019-03-19 | 2022-01-13 | President and Fellows of Harvard College | Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen |
| KR20220017887A (ko) * | 2019-03-27 | 2022-02-14 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Cas9/rnp 및 aav 바이러스의 조합을 사용한 암 면역요법을 위한 키메라 항원 수용체 (car)-일차 nk 세포의 생성 |
| KR20210113393A (ko) * | 2019-03-28 | 2021-09-15 | 주식회사 툴젠 | 혈액응고인자 viii 유전자 역위 보정에 의한 혈우병 치료용 조성물 |
| KR20220009950A (ko) * | 2019-04-15 | 2022-01-25 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 효소 발현의 조절 및 자가-불활성화를 위한 조성물 그리고 효소의 오프-타깃 활동성의 조정 방법 |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2021072328A1 (en) | 2019-10-10 | 2021-04-15 | The Broad Institute, Inc. | Methods and compositions for prime editing rna |
| CN111370055B (zh) * | 2020-03-05 | 2023-05-23 | 中南大学 | 内含子保留预测模型建立方法及其预测方法 |
| WO2021195218A1 (en) * | 2020-03-24 | 2021-09-30 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics |
| 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 |
| WO2022170059A1 (en) | 2021-02-05 | 2022-08-11 | Christiana Care Health Services, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| EP4301462A1 (en) * | 2021-03-04 | 2024-01-10 | Research Institute at Nationwide Children's Hospital | Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications |
| US20220325298A1 (en) * | 2021-04-12 | 2022-10-13 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Crispr epigenetic therapeutics for pain management |
| JP7780172B2 (ja) * | 2021-07-30 | 2025-12-04 | 国立研究開発法人理化学研究所 | 筋萎縮性側索硬化症におけるｃｅｐ２９０遺伝子の変異 |
| CN114306277B (zh) * | 2021-11-02 | 2023-08-04 | 中国人民解放军空军军医大学 | 一种靶向psma的巨噬细胞膜包裹的仿生纳米药物载体及应用 |
| EP4223877A1 (en) | 2022-02-08 | 2023-08-09 | Eberhard Karls Universität Tübingen Medizinische Fakultät | System and method for editing genomic dna to modulate splicing |
| WO2025135916A1 (ko) * | 2023-12-22 | 2025-06-26 | 주식회사 툴젠 | 낮은 면역원성을 가지는 cas9 단백질 변이체 |
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-
2016
- 2016-04-15 WO PCT/US2016/027987 patent/WO2016186772A2/en not_active Ceased
- 2016-04-15 KR KR1020237033136A patent/KR20230142649A/ko active Pending
- 2016-04-15 BR BR112017024514-0A patent/BR112017024514A2/pt active Search and Examination
- 2016-04-15 SG SG10201912907PA patent/SG10201912907PA/en unknown
- 2016-04-15 EP EP16718840.8A patent/EP3298134B8/en active Active
- 2016-04-15 IL IL296365A patent/IL296365A/en unknown
- 2016-04-15 CN CN202210293007.1A patent/CN114657180A/zh active Pending
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