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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- 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 (3) | CN109153994A (ja) |
| AU (2) | AU2017235333B2 (ja) |
| CA (1) | CA3017956A1 (ja) |
| IL (1) | IL261714A (ja) |
| MX (1) | MX2018011114A (ja) |
| SG (1) | SG11201807859WA (ja) |
| WO (1) | WO2017160890A1 (ja) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
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| EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | 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 |
| AU2016261358B2 (en) | 2015-05-11 | 2021-09-16 | Editas Medicine, Inc. | Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells |
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| 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 |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| JP7305539B2 (ja) * | 2016-11-02 | 2023-07-10 | ウニベルシテート バーゼル | 細胞療法に使用するための、免疫学的に識別できる細胞表面変異体 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | 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 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| WO2018170184A1 (en) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| EP3615664A4 (en) * | 2017-04-24 | 2021-01-27 | Seattle Children's Hospital (DBA Seattle Children's Research Institute) | HOMOLOGY-BASED REPAIR COMPOSITIONS FOR THE TREATMENT OF HEMOGLOBINOPATHIES |
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| 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 |
| US20200140896A1 (en) * | 2017-06-30 | 2020-05-07 | Novartis Ag | Methods for the treatment of disease with gene editing systems |
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| 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) |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| CA3082251A1 (en) * | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| US20210180091A1 (en) * | 2017-10-26 | 2021-06-17 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| US20230137729A1 (en) * | 2017-11-06 | 2023-05-04 | Editas Medicine, Inc. | Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy |
| CA3085338A1 (en) * | 2017-12-11 | 2019-06-20 | Editas Medicine, Inc. | Cpf1-related methods and compositions for gene editing |
| 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 |
| EP3765617A1 (en) * | 2018-03-14 | 2021-01-20 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| SG11202008956XA (en) * | 2018-03-14 | 2020-10-29 | Editas Medicine Inc | Systems and methods for the treatment of hemoglobinopathies |
| US20200149070A1 (en) * | 2018-04-24 | 2020-05-14 | Ligandal, Inc. | Methods and compositions for genome editing |
| US20220033856A1 (en) * | 2018-09-11 | 2022-02-03 | Université de Paris | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
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| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| 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 |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix 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 |
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| DK3066201T3 (en) * | 2013-11-07 | 2018-06-06 | Editas Medicine Inc | CRISPR-RELATED PROCEDURES AND COMPOSITIONS WITH LEADING GRADES |
| EP3553176A1 (en) | 2014-03-10 | 2019-10-16 | 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 |
| WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods 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 |
| AU2016261358B2 (en) | 2015-05-11 | 2021-09-16 | Editas Medicine, Inc. | Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells |
-
2017
- 2017-03-14 EP EP17713843.5A patent/EP3430142A1/en active Pending
- 2017-03-14 SG SG11201807859WA patent/SG11201807859WA/en unknown
- 2017-03-14 CN CN201780029929.9A patent/CN109153994A/zh active Pending
- 2017-03-14 US US16/085,480 patent/US20200255857A1/en not_active Abandoned
- 2017-03-14 MX MX2018011114A patent/MX2018011114A/es unknown
- 2017-03-14 KR KR1020237015832A patent/KR20230070331A/ko not_active Application Discontinuation
- 2017-03-14 AU AU2017235333A patent/AU2017235333B2/en active Active
- 2017-03-14 JP JP2018548318A patent/JP2019508051A/ja active Pending
- 2017-03-14 WO PCT/US2017/022377 patent/WO2017160890A1/en active Application Filing
- 2017-03-14 CN CN202311860322.9A patent/CN117821458A/zh active Pending
- 2017-03-14 KR KR1020187029140A patent/KR102532663B1/ko active IP Right Grant
- 2017-03-14 CN CN202311860310.6A patent/CN117802102A/zh active Pending
- 2017-03-14 CA CA3017956A patent/CA3017956A1/en active Pending
-
2018
- 2018-09-12 IL IL261714A patent/IL261714A/en unknown
-
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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