JP2019508051A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- cells
- cell
- grna
- targeting domain
- 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
- 210000004027 cell Anatomy 0.000 claims 13
- 239000002773 nucleotide Substances 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- 108020005004 Guide RNA Proteins 0.000 claims 9
- 238000000034 method Methods 0.000 claims 9
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 7
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 7
- 210000005260 human cell Anatomy 0.000 claims 7
- 230000000295 complement effect Effects 0.000 claims 6
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 230000008685 targeting Effects 0.000 claims 6
- 238000010362 genome editing Methods 0.000 claims 5
- 210000003743 erythrocyte Anatomy 0.000 claims 4
- 210000003924 normoblast Anatomy 0.000 claims 4
- 239000002243 precursor Substances 0.000 claims 4
- 108091033409 CRISPR Proteins 0.000 claims 3
- 102100038614 Hemoglobin subunit gamma-1 Human genes 0.000 claims 3
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims 3
- 101001031977 Homo sapiens Hemoglobin subunit gamma-1 Proteins 0.000 claims 3
- 101001031961 Homo sapiens Hemoglobin subunit gamma-2 Proteins 0.000 claims 3
- 238000006640 acetylation reaction Methods 0.000 claims 3
- 238000007069 methylation reaction Methods 0.000 claims 3
- 230000001105 regulatory effect 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 cell Anatomy 0.000 claims 2
- 230000007774 longterm Effects 0.000 claims 2
- 108010044495 Fetal Hemoglobin Proteins 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 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
- 229940046166 oligodeoxynucleotide Drugs 0.000 claims 1
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 (enExample) | 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 (2) | US20200255857A1 (enExample) |
| EP (1) | EP3430142A1 (enExample) |
| JP (2) | JP2019508051A (enExample) |
| KR (2) | KR102532663B1 (enExample) |
| CN (4) | CN117821458A (enExample) |
| AU (2) | AU2017235333B2 (enExample) |
| CA (1) | CA3017956A1 (enExample) |
| IL (2) | IL261714B2 (enExample) |
| MX (2) | MX2018011114A (enExample) |
| SG (1) | SG11201807859WA (enExample) |
| WO (1) | WO2017160890A1 (enExample) |
Families Citing this family (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | 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 |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| EP3262173A2 (en) | 2015-02-23 | 2018-01-03 | Crispr Therapeutics AG | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| AU2016225179C1 (en) | 2015-02-23 | 2022-11-03 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| JP7030522B2 (ja) | 2015-05-11 | 2022-03-07 | エディタス・メディシン、インコーポレイテッド | 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法 |
| CN108026526B (zh) | 2015-06-09 | 2023-05-12 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| WO2017077394A2 (en) | 2015-11-04 | 2017-05-11 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | 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 |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | 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 | 核酸塩基エディターのａａｖ送達 |
| 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 |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| 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 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| BR112019019655A2 (pt) | 2017-03-23 | 2020-04-22 | Harvard College | editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico |
| WO2018200597A1 (en) | 2017-04-24 | 2018-11-01 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Homology directed repair compositions for the treatment of hemoglobinopathies |
| 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 |
| WO2019003193A1 (en) * | 2017-06-30 | 2019-01-03 | Novartis Ag | METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS |
| 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 |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019081982A1 (en) * | 2017-10-26 | 2019-05-02 | Crispr Therapeutics Ag | SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES |
| WO2019090202A1 (en) * | 2017-11-06 | 2019-05-09 | Editas Medicine, Inc. | Methods, compositions and components for crispr-cas9 editing of cblb in t cells for immunotherapy |
| MA51107A (fr) | 2017-12-05 | 2020-10-14 | Vertex Pharma | Cellules souches et progénitrices hématopoïétiques humaines cd34+ modifiées par crispr-cas9 et utilisations associées |
| EP3724326A1 (en) * | 2017-12-11 | 2020-10-21 | Editas Medicine, Inc. | Cpf1-related methods and compositions for gene editing |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| WO2019150203A1 (en) | 2018-02-05 | 2019-08-08 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| CA3093289A1 (en) * | 2018-03-07 | 2019-09-12 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| CA3093702A1 (en) | 2018-03-14 | 2019-09-19 | 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 |
| EP3796894A4 (en) * | 2018-04-24 | 2022-05-04 | Ligandal, Inc. | METHODS AND COMPOSITIONS FOR GENOMIC EDITING |
| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク． | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| 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 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| EP3887521A1 (en) * | 2018-11-29 | 2021-10-06 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| KR20210102309A (ko) * | 2018-12-05 | 2021-08-19 | 프레드 헛친슨 켄서 리서치 센터 | 유전적으로-변형된 세포를 제작하는 줄어든, 그리고 최소한의 조작 |
| 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 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN116497067B (zh) | 2019-02-13 | 2025-01-24 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| 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 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| 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 |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | 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 |
| EP4409004A1 (en) * | 2021-11-02 | 2024-08-07 | 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 |
| WO2025038164A1 (en) * | 2023-08-15 | 2025-02-20 | University Of Massachusetts | Nick resection in cancer |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
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 |
| SG10201606959PA (en) * | 2012-02-24 | 2016-09-29 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| SG11201500852WA (en) * | 2012-08-29 | 2015-04-29 | Sangamo Biosciences 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 |
| HK1223401A1 (zh) * | 2013-05-15 | 2017-07-28 | 桑格摩生物科学股份有限公司 | 用於治疗遗传病状的方法和组合物 |
| KR20230136697A (ko) * | 2013-06-05 | 2023-09-26 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
| CA2930015A1 (en) * | 2013-11-07 | 2015-05-14 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| WO2015148863A2 (en) * | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP4464338A3 (en) | 2014-11-07 | 2025-02-12 | Editas Medicine, Inc. | Systems for improving crispr/cas-mediated genome-editing |
| AU2016225179C1 (en) * | 2015-02-23 | 2022-11-03 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| JP7030522B2 (ja) | 2015-05-11 | 2022-03-07 | エディタス・メディシン、インコーポレイテッド | 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法 |
-
2017
- 2017-03-14 JP JP2018548318A patent/JP2019508051A/ja active Pending
- 2017-03-14 KR KR1020187029140A patent/KR102532663B1/ko active Active
- 2017-03-14 CN CN202311860322.9A patent/CN117821458A/zh active Pending
- 2017-03-14 WO PCT/US2017/022377 patent/WO2017160890A1/en not_active Ceased
- 2017-03-14 SG SG11201807859WA patent/SG11201807859WA/en unknown
- 2017-03-14 CA CA3017956A patent/CA3017956A1/en 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 IL IL261714A patent/IL261714B2/en unknown
- 2017-03-14 CN CN202311860310.6A patent/CN117802102A/zh active Pending
- 2017-03-14 IL IL313038A patent/IL313038A/en unknown
- 2017-03-14 EP EP17713843.5A patent/EP3430142A1/en active Pending
- 2017-03-14 CN CN202311860300.2A patent/CN118127006A/zh active Pending
- 2017-03-14 CN CN201780029929.9A patent/CN109153994A/zh active Pending
- 2017-03-14 KR KR1020237015832A patent/KR102723347B1/ko active Active
- 2017-03-14 AU AU2017235333A patent/AU2017235333B2/en active Active
-
2018
- 2018-09-13 MX MX2024012899A patent/MX2024012899A/es unknown
-
2023
- 2023-02-24 JP JP2023026918A patent/JP2023075166A/ja active Pending
- 2023-08-08 AU AU2023214243A patent/AU2023214243A1/en active Pending
- 2023-08-28 US US18/457,258 patent/US20240360471A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019508051A5 (enExample) | ||
| JP7698828B2 (ja) | Ｒｎａを編集する方法および組成物 | |
| Pham | Medical biotechnology: techniques and applications | |
| JP7197363B2 (ja) | ヌクレアーゼを使用するヒト神経幹細胞のゲノム編集 | |
| MX2024006956A (es) | Inserciones de adn no virales orientadas. | |
| FI3445388T3 (fi) | Materiaaleja ja menetelmiä hemoglobinopatioiden hoitamiseksi | |
| JP2016500262A5 (enExample) | ||
| MX2018008733A (es) | Metodos para extender la capacidad replicativa de las celulas somaticas durante un proceso de cultivo ex vivo. | |
| EA201791991A1 (ru) | Способ улучшения способности противодействовать внедренным днк-содержащим вирусам растения | |
| WO2021076744A1 (en) | Gene targets for manipulating t cell behavior | |
| JP2016512691A5 (enExample) | ||
| MX2018001617A (es) | Composiciones de crispr-cas9 diseñados y metodos de uso. | |
| WO2019103442A3 (ko) | CRISPR/Cpf1 시스템을 이용한 유전체 편집용 조성물 및 이의 용도 | |
| CN106554969A (zh) | 基于抑菌杀菌的多靶点CRISPR/Cas9表达载体 | |
| JP2020524998A5 (enExample) | ||
| AU2016381313A1 (en) | Compositions and methods for the treatment of hemoglobinopathies | |
| JP2015533786A5 (enExample) | ||
| HRP20211468T1 (hr) | Postupci i pripravci za nukleazno posredovani inženjering genoma i ispravak u krvotvornim matičnim stanicama | |
| RU2022103641A (ru) | Искусственная модификация генома для регуляции экспрессии гена | |
| MX2021004455A (es) | Composiciones y métodos para administrar transgenes. | |
| CN106957857A (zh) | 一种利用CRISPR/Cas9系统共同敲除山羊MSTN和FGF5基因的方法 | |
| Matsunaga et al. | Single-step generation of gene knockout-rescue system in pluripotent stem cells by promoter insertion with CRISPR/Cas9 | |
| Ran | CRISPR-Cas: Development and applications for mammalian genome editing | |
| Zhu et al. | Gene targeting through homologous recombination in monkey embryonic stem cells using CRISPR/Cas9 system | |
| DeWitt et al. | Efficient correction of the sickle mutation in human hematopoietic stem cells using a Cas9 ribonucleoprotein complex |