JP2020517270A5 - - Google Patents
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- Publication number
- JP2020517270A5 JP2020517270A5 JP2019557380A JP2019557380A JP2020517270A5 JP 2020517270 A5 JP2020517270 A5 JP 2020517270A5 JP 2019557380 A JP2019557380 A JP 2019557380A JP 2019557380 A JP2019557380 A JP 2019557380A JP 2020517270 A5 JP2020517270 A5 JP 2020517270A5
- Authority
- JP
- Japan
- Prior art keywords
- cells
- nuclease
- nucleic acid
- cell
- binding site
- 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
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- 210000004027 cells Anatomy 0.000 claims 19
- 101700080605 NUC1 Proteins 0.000 claims 11
- 101700006494 nucA Proteins 0.000 claims 11
- 150000007523 nucleic acids Chemical class 0.000 claims 11
- 108020004707 nucleic acids Proteins 0.000 claims 10
- 102000001392 Wiskott-Aldrich Syndrome Protein Human genes 0.000 claims 8
- 108010093528 Wiskott-Aldrich Syndrome Protein Proteins 0.000 claims 8
- 210000003958 Hematopoietic Stem Cells Anatomy 0.000 claims 6
- 208000006110 Wiskott-Aldrich Syndrome Diseases 0.000 claims 6
- 229920000023 polynucleotide Polymers 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 241000432074 Adeno-associated virus Species 0.000 claims 2
- 102100016492 CD34 Human genes 0.000 claims 2
- 108060001251 CD34 Proteins 0.000 claims 2
- 229920000033 CRISPR Polymers 0.000 claims 2
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 2
- 210000001744 T-Lymphocytes Anatomy 0.000 claims 2
- 238000002744 homologous recombination Methods 0.000 claims 2
- 241000219430 Betula pendula Species 0.000 claims 1
- 238000010354 CRISPR gene editing Methods 0.000 claims 1
- 108010082319 CRISPR-Associated Protein 9 Proteins 0.000 claims 1
- 229920002391 Guide RNA Polymers 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 208000007617 Thrombocytopenia 1 Diseases 0.000 claims 1
- 230000000996 additive Effects 0.000 claims 1
- 239000000654 additive Substances 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 230000002708 enhancing Effects 0.000 claims 1
- 230000003394 haemopoietic Effects 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
- 230000035897 transcription Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762488249P | 2017-04-21 | 2017-04-21 | |
| US62/488,249 | 2017-04-21 | ||
| PCT/US2018/028442 WO2018195360A1 (en) | 2017-04-21 | 2018-04-19 | Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020517270A JP2020517270A (ja) | 2020-06-18 |
| JP2020517270A5 true JP2020517270A5 (US07794700-20100914-C00152.png) | 2021-06-10 |
Family
ID=63856121
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019557380A Pending JP2020517270A (ja) | 2017-04-21 | 2018-04-19 | ウィスコット・アルドリッチ症候群およびｘ連鎖性血小板減少症の治療的ゲノム編集 |
Country Status (7)
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020150499A1 (en) * | 2019-01-16 | 2020-07-23 | Fred Hutchinson Cancer Research Center | Methods to tag and isolate cells infected with the human immunodeficiency virus |
| WO2021096887A1 (en) * | 2019-11-11 | 2021-05-20 | The Regents Of The University Of California | Lentiviral vectors in hematopoietic stem cells to treat wiskott-aldrich syndrome (was) |
| CN111808859B (zh) * | 2020-07-13 | 2022-09-13 | 中国科学院广州生物医药与健康研究院 | WAS基因的gRNA及其应用 |
| CN115927585B (zh) * | 2022-08-29 | 2024-04-23 | 湖南家辉生物技术有限公司 | WAS致病突变基因及在制备Wiskott-Aldrich综合征诊断试剂盒中的应用 |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7582738B2 (en) * | 2004-12-17 | 2009-09-01 | Children's Hospital Medical Center | Diagnostic assay for Wiskott-Aldrich syndrome and genetically related disorders |
| US20160237455A1 (en) * | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| DK3441468T3 (da) * | 2013-10-17 | 2021-07-26 | Sangamo Therapeutics Inc | Afgivelsesfremgangsmåder og sammensætninger til nukleasemedieret genom-manipulation |
| AU2015330699B2 (en) | 2014-10-10 | 2021-12-02 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| JP7059179B2 (ja) * | 2015-10-20 | 2022-04-25 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | 遺伝子操作のための方法及び製品 |
| US20180127786A1 (en) * | 2016-09-23 | 2018-05-10 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
| US20200206322A1 (en) * | 2017-03-21 | 2020-07-02 | Stitch Bio, Llc | Treating cancer with cas endonuclease complexes |
| CN112469823A (zh) * | 2018-04-27 | 2021-03-09 | 西雅图儿童医院d/b/a西雅图儿童研究所 | 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑 |
| KR20210005178A (ko) | 2018-04-27 | 2021-01-13 | 시애틀 칠드런즈 호스피탈 디/비/에이 시애틀 칠드런즈 리서치 인스티튜트 | X-연관 고 igm 증후군에서의 치료적 게놈 편집 |
| US11521964B2 (en) | 2018-06-29 | 2022-12-06 | Intel Corporation | Schottky diode structures and integration with III-V transistors |
-
2018
- 2018-04-19 CN CN201880041482.1A patent/CN110785179A/zh active Pending
- 2018-04-19 EP EP18788173.5A patent/EP3612194A4/en active Pending
- 2018-04-19 CA CA3060570A patent/CA3060570A1/en active Pending
- 2018-04-19 US US16/605,748 patent/US11643671B2/en active Active
- 2018-04-19 AU AU2018254529A patent/AU2018254529B2/en active Active
- 2018-04-19 JP JP2019557380A patent/JP2020517270A/ja active Pending
- 2018-04-19 WO PCT/US2018/028442 patent/WO2018195360A1/en active Application Filing
-
2023
- 2023-04-05 US US18/296,235 patent/US20240141389A1/en active Pending
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