JP2019508051A5 - - Google Patents
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- JP2019508051A5 JP2019508051A5 JP2018548318A JP2018548318A JP2019508051A5 JP 2019508051 A5 JP2019508051 A5 JP 2019508051A5 JP 2018548318 A JP2018548318 A JP 2018548318A JP 2018548318 A JP2018548318 A JP 2018548318A JP 2019508051 A5 JP2019508051 A5 JP 2019508051A5
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- nucleotide sequence
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- 210000004027 cells Anatomy 0.000 claims 20
- 229920002391 Guide RNA Polymers 0.000 claims 9
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 9
- 108020005004 Guide RNA Proteins 0.000 claims 8
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 7
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 7
- 101700022675 HBG Proteins 0.000 claims 6
- 230000000295 complement Effects 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 238000006011 modification reaction Methods 0.000 claims 6
- 238000010362 genome editing Methods 0.000 claims 5
- 210000004494 Erythroblasts Anatomy 0.000 claims 4
- 210000003743 Erythrocytes Anatomy 0.000 claims 4
- 102000014961 Protein Precursors Human genes 0.000 claims 4
- 108010078762 Protein Precursors Proteins 0.000 claims 4
- OZAIFHULBGXAKX-UHFFFAOYSA-N precursor Substances N#CC(C)(C)N=NC(C)(C)C#N OZAIFHULBGXAKX-UHFFFAOYSA-N 0.000 claims 4
- 108010082319 CRISPR-Associated Protein 9 Proteins 0.000 claims 3
- 101700073128 HBG1 Proteins 0.000 claims 3
- 102100004900 HBG1 Human genes 0.000 claims 3
- 101700075536 HBG2 Proteins 0.000 claims 3
- 102100004899 HBG2 Human genes 0.000 claims 3
- 238000006640 acetylation reaction Methods 0.000 claims 3
- 238000007069 methylation reaction Methods 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 230000001105 regulatory Effects 0.000 claims 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 210000003958 Hematopoietic Stem Cells Anatomy 0.000 claims 2
- 102100016492 CD34 Human genes 0.000 claims 1
- 108060001251 CD34 Proteins 0.000 claims 1
- 108010044495 Fetal Hemoglobin Proteins 0.000 claims 1
- 238000004581 coalescence Methods 0.000 claims 1
- 238000004520 electroporation Methods 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
Claims (14)
- ゲノム編集によってヒト細胞の胎児ヘモグロビンレベルを増加させる、インビトロまたはエクスビボで行う方法であって、前記方法は前記ヒト細胞にリボ核タンパク質（ＲＮＰ）複合体を導入するステップを含み、
前記リボ核タンパク質複合体が：
（ａ）Ｃａｓ９エンドヌクレアーゼタンパク質；および
（ｂ）ＨＢＧ１またはＨＢＧ２調節領域内に完全にまたは部分的に位置する標的ドメインに相補的または部分的に相補的なヌクレオチド配列を含む標的化ドメインを含むガイドＲＮＡ（ｇＲＮＡ）を含む、方法。 - 前記標的化ドメインが、配列番号２５１〜９０１のいずれか１つに記載のヌクレオチド配列と同一であるか、または１、２、３、４、もしくは５個以下のヌクレオチドのみ異なるヌクレオチド配列を含む、請求項１に記載の方法。
- 前記ヒト細胞が、（１）赤芽球に分化することができる細胞、（２）赤血球に分化することができる細胞、（３）赤芽球の前駆体、（４）赤血球の前駆体、および（５）長期造血幹細胞（ＬＴ−ＨＳＣ）からなる群から選択される１つ以上の細胞から選択される、請求項１に記載の方法。
- 前記ヒト細胞がＣＤ３４＋細胞である、請求項３に記載の方法。
- 前記ｇＲＮＡが、２’−アセチル化、２’−メチル化、およびホスホロチオエート修飾からなる群から選択される１つ以上の修飾を含む、請求項１に記載の方法。
- 前記ヒト細胞に導入するステップが、前記ヒト細胞に一本鎖オリゴデオキシヌクレオチド（ｓｓＯＤＮ）を導入することをさらに含む、請求項１に記載の方法。
- 前記ヒト細胞に導入するステップが、電気穿孔によって行われる、請求項１に記載の方法。
- リボ核タンパク質（ＲＮＰ）複合体を用いて修飾された細胞であって：
（ａ）Ｃａｓ９エンドヌクレアーゼタンパク質；および
（ｂ）ＨＢＧ１またはＨＢＧ２調節領域内に完全にまたは部分的に位置する標的ドメインに相補的または部分的に相補的なヌクレオチド配列を含む標的化ドメインを含むガイドＲＮＡ（ｇＲＮＡ）を含む、細胞。 - 前記標的化ドメインが、配列番号２５１〜９０１のいずれか１つに記載のヌクレオチド配列と同一であるか、または１、２、３、４、もしくは５個以下のヌクレオチドのみ異なるヌクレオチド配列を含む、請求項８に記載の細胞。
- 前記ｇＲＮＡが、２’−アセチル化、２’−メチル化、およびホスホロチオエート修飾からなる群から選択される１つ以上の修飾を含む、請求項８に記載の細胞。
- 前記細胞が、（１）赤芽球に分化することができる細胞、（２）赤血球に分化することができる細胞、（３）赤芽球の前駆体、（４）赤血球の前駆体、および（５）長期造血幹細胞（ＬＴ−ＨＳＣ）からなる群から選択される１つ以上の細胞から選択される、請求項８に記載の細胞。
- リボ核タンパク質（ＲＮＰ）複合体を含むゲノム編集システムであって：
（ａ）Ｃａｓ９エンドヌクレアーゼタンパク質；および
（ｂ）ＨＢＧ１またはＨＢＧ２調節領域内に完全にまたは部分的に位置する標的ドメインに相補的または部分的に相補的なヌクレオチド配列を含む標的化ドメインを含むガイドＲＮＡ（ｇＲＮＡ）を含む、ゲノム編集システム。 - 前記標的化ドメインが、配列番号２５１〜９０１のいずれか１つに記載のヌクレオチド配列と同一であるか、または１、２、３、４、もしくは５個以下のヌクレオチドのみ異なるヌクレオチド配列を含む、請求項１２に記載のゲノム編集システム。
- 前記ｇＲＮＡが、２’−アセチル化、２’−メチル化、およびホスホロチオエート修飾からなる群から選択される１つ以上の修飾を含む、請求項１２に記載のゲノム編集システム。
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023026918A JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662308190P | 2016-03-14 | 2016-03-14 | |
| US62/308,190 | 2016-03-14 | ||
| US201762456615P | 2017-02-08 | 2017-02-08 | |
| US62/456,615 | 2017-02-08 | ||
| PCT/US2017/022377 WO2017160890A1 (en) | 2016-03-14 | 2017-03-14 | Crispr/cas-related methods and compositions for treating beta hemoglobinopathies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026918A Division JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019508051A JP2019508051A (ja) | 2019-03-28 |
| JP2019508051A5 true JP2019508051A5 (ja) | 2020-04-23 |
Family
ID=58413206
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018548318A Pending JP2019508051A (ja) | 2016-03-14 | 2017-03-14 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
| JP2023026918A Pending JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026918A Pending JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20200255857A1 (ja) |
| EP (1) | EP3430142A1 (ja) |
| JP (2) | JP2019508051A (ja) |
| KR (2) | KR20230070331A (ja) |
| CN (4) | CN117802102A (ja) |
| AU (2) | AU2017235333B2 (ja) |
| CA (1) | CA3017956A1 (ja) |
| IL (2) | IL313038A (ja) |
| MX (1) | MX2018011114A (ja) |
| SG (1) | SG11201807859WA (ja) |
| WO (1) | WO2017160890A1 (ja) |
Families Citing this family (52)
| 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 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| 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 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| BR112017017812A2 (pt) | 2015-02-23 | 2018-04-10 | Crispr Therapeutics Ag | materiais e métodos para tratamento de hemoglobinopatias |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| AU2016276702B2 (en) | 2015-06-09 | 2022-07-28 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for improving transplantation |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | 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 | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| US12043843B2 (en) | 2015-11-04 | 2024-07-23 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| SG11201903089RA (en) | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| KR20240043810A (ko) * | 2016-11-02 | 2024-04-03 | 유니버시타트 바셀 | 세포 치료에 사용하기 위한 면역학적으로 식별 가능한 세포 표면 변이체 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| 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 |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| IL306092A (en) | 2017-03-23 | 2023-11-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
| US20210222201A1 (en) * | 2017-04-24 | 2021-07-22 | Seattie Children's Hospital (dba Seattle Children's Research Institute) | Homology directed repair compositions for the treatment of hemoglobinopathies |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | 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 |
| US20200140896A1 (en) * | 2017-06-30 | 2020-05-07 | Novartis Ag | Methods for the treatment of disease with gene editing systems |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | 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) |
| EP3676376A2 (en) | 2017-08-30 | 2020-07-08 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| CN111757937A (zh) * | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| EP3701029A1 (en) * | 2017-10-26 | 2020-09-02 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| JP2021502077A (ja) * | 2017-11-06 | 2021-01-28 | エディタス・メディシン，インコーポレイテッド | 免疫療法のためのｔ細胞におけるｃｂｌｂのｃｒｉｓｐｒ−ｃａｓ９編集のための方法、組成物および構成要素 |
| CN111712569A (zh) * | 2017-12-11 | 2020-09-25 | 爱迪塔斯医药公司 | 用于基因编辑的Cpf1-相关方法和组合物 |
| US11268077B2 (en) | 2018-02-05 | 2022-03-08 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| AU2019230210A1 (en) * | 2018-03-07 | 2020-10-01 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| AU2019236209A1 (en) * | 2018-03-14 | 2020-10-01 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| AU2019234922A1 (en) * | 2018-03-14 | 2020-10-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2019210005A1 (en) * | 2018-04-24 | 2019-10-31 | Ligandal, Inc. | Methods and compositions for genome editing |
| EP3850094A1 (en) * | 2018-09-11 | 2021-07-21 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
| US20220047637A1 (en) * | 2018-11-29 | 2022-02-17 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| 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 |
| CA3128755C (en) | 2019-02-13 | 2024-06-04 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| WO2020191241A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| CN112011576A (zh) * | 2019-05-31 | 2020-12-01 | 华东师范大学 | Crispr基因编辑技术在治疗地中海贫血中的应用 |
| CN112979823B (zh) * | 2019-12-18 | 2022-04-08 | 华东师范大学 | 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白 |
| 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 |
| CN111876416B (zh) * | 2020-07-01 | 2021-09-03 | 广州瑞风生物科技有限公司 | 激活γ-珠蛋白基因表达的方法和组合物 |
| WO2023079465A1 (en) * | 2021-11-02 | 2023-05-11 | The University Of British Columbia | Compositions and methods for preventing, ameliorating, or treating sickle cell disease |
| CN114848851A (zh) * | 2022-04-29 | 2022-08-05 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 治疗β-地中海贫血的药物 |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| KR101833589B1 (ko) * | 2012-02-24 | 2018-03-02 | 프레드 헛친슨 켄서 리서치 센터 | 이상혈색소증 치료를 위한 조성물 및 방법 |
| AU2013308770B2 (en) * | 2012-08-29 | 2019-01-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| EP2925864B1 (en) * | 2012-11-27 | 2018-10-31 | The Children's Medical Center Corporation | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
| CN116083487A (zh) * | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| EP4389903A2 (en) * | 2013-06-05 | 2024-06-26 | Duke University | Rna-guided gene editing and gene regulation |
| PT3066201T (pt) * | 2013-11-07 | 2018-06-04 | Massachusetts Inst Technology | Métodos e composições relacionadas com crispr com arng reguladores |
| EP3116997B1 (en) | 2014-03-10 | 2019-05-15 | 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 |
| EP3215617B1 (en) | 2014-11-07 | 2024-05-08 | Editas Medicine, Inc. | Systems for improving crispr/cas-mediated genome-editing |
| BR112017017812A2 (pt) * | 2015-02-23 | 2018-04-10 | Crispr Therapeutics Ag | materiais e métodos para tratamento de hemoglobinopatias |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
-
2017
- 2017-03-14 CN CN202311860310.6A patent/CN117802102A/zh active Pending
- 2017-03-14 MX MX2018011114A patent/MX2018011114A/es unknown
- 2017-03-14 CN CN201780029929.9A patent/CN109153994A/zh active Pending
- 2017-03-14 CN CN202311860300.2A patent/CN118127006A/zh active Pending
- 2017-03-14 AU AU2017235333A patent/AU2017235333B2/en active Active
- 2017-03-14 SG SG11201807859WA patent/SG11201807859WA/en unknown
- 2017-03-14 IL IL313038A patent/IL313038A/en unknown
- 2017-03-14 CA CA3017956A patent/CA3017956A1/en active Pending
- 2017-03-14 WO PCT/US2017/022377 patent/WO2017160890A1/en active Application Filing
- 2017-03-14 JP JP2018548318A patent/JP2019508051A/ja active Pending
- 2017-03-14 EP EP17713843.5A patent/EP3430142A1/en active Pending
- 2017-03-14 CN CN202311860322.9A patent/CN117821458A/zh active Pending
- 2017-03-14 US US16/085,480 patent/US20200255857A1/en not_active Abandoned
- 2017-03-14 KR KR1020237015832A patent/KR20230070331A/ko not_active Application Discontinuation
- 2017-03-14 IL IL261714A patent/IL261714B1/en unknown
- 2017-03-14 KR KR1020187029140A patent/KR102532663B1/ko active IP Right Grant
-
2023
- 2023-02-24 JP JP2023026918A patent/JP2023075166A/ja active Pending
- 2023-08-08 AU AU2023214243A patent/AU2023214243A1/en active Pending
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