JP2021521855A - ヘモグロビン関連変異の編集のための相同組換え修復用鋳型の設計およびその送達 - Google Patents

ヘモグロビン関連変異の編集のための相同組換え修復用鋳型の設計およびその送達 Download PDF

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JP2021521855A
JP2021521855A JP2020560396A JP2020560396A JP2021521855A JP 2021521855 A JP2021521855 A JP 2021521855A JP 2020560396 A JP2020560396 A JP 2020560396A JP 2020560396 A JP2020560396 A JP 2020560396A JP 2021521855 A JP2021521855 A JP 2021521855A
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cell
cells
nucleic acid
nuclease
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ジェイ． ローリングス，デイヴィッド
ジェイ． ローリングス，デイヴィッド
パタビ，ソウミャ
エム． シャーレンバーグ，アンドリュー
エム． シャーレンバーグ，アンドリュー
ヤコビ，カイル
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シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート）
シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート）
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JP2020560396A 2018-04-27 2019-04-24 ヘモグロビン関連変異の編集のための相同組換え修復用鋳型の設計およびその送達 Pending JP2021521855A (ja)

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US201862663553P 2018-04-27 2018-04-27
US62/663,553 2018-04-27
US201962820521P 2019-03-19 2019-03-19
US62/820,521 2019-03-19
PCT/US2019/028861 WO2019209914A2 (en) 2018-04-27 2019-04-24 Homology-directed repair template design and delivery to edit hemoglobin-related mutations

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JP2021521855A true JP2021521855A (ja) 2021-08-30
JP2021521855A5 JP2021521855A5 (https=) 2023-03-20

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US (1) US20210309995A1 (https=)
JP (1) JP2021521855A (https=)
KR (1) KR20210005179A (https=)
CN (1) CN112313334A (https=)
AU (1) AU2019261387A1 (https=)
CA (1) CA3106812A1 (https=)
WO (1) WO2019209914A2 (https=)

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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
CN109486814A (zh) * 2017-10-31 2019-03-19 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
US20220251566A1 (en) * 2019-06-26 2022-08-11 The Research Foundation For The State University Of New York Cells engineered for oligonucleotide delivery, and methods for making and using thereof
CN113186203B (zh) * 2020-02-13 2022-12-30 斯微(上海)生物科技股份有限公司 治疗或者预防冠状病毒病的疫苗试剂
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
WO2022221319A2 (en) * 2021-04-12 2022-10-20 Graphite Bio, Inc. Methods and compositions for production of genetically modified primary cells
CN113174433B (zh) * 2021-04-22 2024-03-26 苏州淦江生物技术有限公司 一种基于Cas蛋白的检测方法
WO2022232839A1 (en) * 2021-04-30 2022-11-03 The Board Of Trustees Of The Leland Stanford Junior University Methods for improved production of primary cd34+ cells
CN115232811B (zh) * 2021-06-28 2025-10-28 南京启真基因工程有限公司 用于构建hbb基因突变的镰刀型细胞贫血症模型猪核移植供体细胞的方法及应用
WO2023275892A1 (en) * 2021-06-29 2023-01-05 Council Of Scientific & Industrial Research Engineered fncas9 and uses thereof
CA3225981A1 (en) * 2021-07-01 2023-01-05 Bluebird Bio, Inc. Methods
WO2023049458A1 (en) * 2021-09-24 2023-03-30 Maxcyte, Inc. A method of enhanced viral transduction using electroporation
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
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
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
WO2025181336A1 (en) * 2024-03-01 2025-09-04 Cellectis Sa Compositions and methods for hbb-editing in hspc

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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
EP3122880B1 (en) * 2014-03-26 2021-05-05 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
JP2018522249A (ja) * 2015-04-24 2018-08-09 エディタス・メディシン、インコーポレイテッド Ｃａｓ９分子／ガイドｒｎａ分子複合体の評価

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US20210309995A1 (en) 2021-10-07
KR20210005179A (ko) 2021-01-13
WO2019209914A2 (en) 2019-10-31
AU2019261387A1 (en) 2020-11-19
WO2019209914A3 (en) 2020-01-16
CA3106812A1 (en) 2019-10-31
EP3784783A2 (en) 2021-03-03

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