KR102912481B1 - 유전자 편집된 t 세포에서의 인간 foxp3의 발현 - Google Patents

유전자 편집된 t 세포에서의 인간 foxp3의 발현

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KR102912481B1
KR102912481B1 KR1020207033790A KR20207033790A KR102912481B1 KR 102912481 B1 KR102912481 B1 KR 102912481B1 KR 1020207033790 A KR1020207033790 A KR 1020207033790A KR 20207033790 A KR20207033790 A KR 20207033790A KR 102912481 B1 KR102912481 B1 KR 102912481B1
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foxp3
sequence
nucleic acid
cells
cell
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KR20210005146A (ko
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앤드류 엠. 샤렌버그
데이비드 제이. 롤링스
카렌 소머
유치 치앙 호나케르
이람 에프. 칸
트로이 토거슨
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시애틀 칠드런즈 호스피탈 디/비/에이 시애틀 칠드런즈 리서치 인스티튜트
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KR1020207033790A 2018-04-27 2019-04-25 유전자 편집된 t 세포에서의 인간 foxp3의 발현 Active KR102912481B1 (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862663561P 2018-04-27 2018-04-27
US62/663,561 2018-04-27
US201862773414P 2018-11-30 2018-11-30
US62/773,414 2018-11-30
PCT/US2019/029159 WO2019210078A1 (en) 2018-04-27 2019-04-25 Expression of human foxp3 in gene edited t cells

Publications (2)

Publication Number Publication Date
KR20210005146A KR20210005146A (ko) 2021-01-13
KR102912481B1 true KR102912481B1 (ko) 2026-01-15

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US (1) US20210253652A1 (enExample)
EP (1) EP3784689A4 (enExample)
JP (3) JP7575949B2 (enExample)
KR (1) KR102912481B1 (enExample)
CN (1) CN112041334A (enExample)
AU (2) AU2019257708B2 (enExample)
CA (1) CA3091491A1 (enExample)
IL (1) IL277036A (enExample)
SG (1) SG11202007877SA (enExample)
WO (1) WO2019210078A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3042179A1 (en) 2016-10-31 2018-05-03 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using cd4 t-cells with engineered stabilization of expression of endogenous foxp3 gene
SG11202007878UA (en) 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells
WO2020161122A1 (en) * 2019-02-05 2020-08-13 INSERM (Institut National de la Santé et de la Recherche Médicale) Recombinant vectors suitable for the treatment of ipex syndrome
JP2022533713A (ja) * 2019-05-21 2022-07-25 サンガモ セラピューティクス， インコーポレイテッド 調節性ｔ細胞における制御された導入遺伝子発現
CA3160113A1 (en) * 2019-11-08 2021-05-14 Sangamo Therapeutics, Inc. Generation of engineered regulatory t cells
EP4103591A4 (en) * 2020-02-13 2024-09-04 The Board of Trustees of the Leland Stanford Junior University Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients
IL302591A (en) * 2020-11-09 2023-07-01 Quell Therapeutics Ltd Method for cryopreservation of transgenic regulatory T cells
WO2022266139A2 (en) * 2021-06-14 2022-12-22 Graphite Bio, Inc. Methods to genetically engineer hematopoietic stem and progenitor cells for red cell specific expression of therapeutic proteins
WO2023122099A2 (en) * 2021-12-21 2023-06-29 The Board Of Trustees Of The Leland Stanford Junior University Crispr-based gene editing to preserve splicing and expression of foxp3 isoforms 1 and 2
CN115305237B (zh) * 2022-07-08 2023-07-14 中南大学 一种优化的调节性t细胞的扩增及规模化生产的方法
WO2024127369A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target foxp3 gene and methods of use
CN121532517A (zh) * 2023-06-24 2026-02-13 索诺玛生物治疗公司 用于对细胞进行工程改造的组合物和方法
CN122055438A (zh) * 2023-09-08 2026-05-15 索诺玛生物治疗公司 异位表达foxp3的细胞
JP7818147B1 (ja) * 2025-01-23 2026-02-20 国立研究開発法人国立がん研究センター エフェクターｔ細胞、およびそれを含む医薬組成物

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016123578A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016183041A2 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110123502A1 (en) 2007-02-21 2011-05-26 Barry Simon C Method for obtaining treg-cells
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
EP3500696A4 (en) 2016-08-16 2020-04-08 Bluebird Bio, Inc. VARIANTS OF IL-10 ALPHA RECEPTOR HOMING ENDONUCLEASE, COMPOSITIONS AND METHODS OF USE
WO2018073391A1 (en) * 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
EP3532075A4 (en) * 2016-10-27 2020-07-08 Intima Bioscience, Inc. VIRAL METHODS OF MANUFACTURING GENETICALLY MODIFIED CELLS
CA3042179A1 (en) 2016-10-31 2018-05-03 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using cd4 t-cells with engineered stabilization of expression of endogenous foxp3 gene
CN109790518A (zh) * 2017-05-08 2019-05-21 中国科学院动物研究所 经修饰的t细胞、其制备方法及用途
WO2019040655A1 (en) * 2017-08-22 2019-02-28 The Regents Of The University Of California LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
SG11202007878UA (en) * 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016123578A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016183041A2 (en) 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods

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US20210253652A1 (en) 2021-08-19
JP7575949B2 (ja) 2024-10-31
JP7844581B2 (ja) 2026-04-13
JP2025013879A (ja) 2025-01-28
AU2019257708B2 (en) 2024-08-22
SG11202007877SA (en) 2020-09-29
AU2024266825A1 (en) 2024-12-12
EP3784689A1 (en) 2021-03-03
CA3091491A1 (en) 2019-10-31
CN112041334A (zh) 2020-12-04
WO2019210078A1 (en) 2019-10-31
KR20210005146A (ko) 2021-01-13
EP3784689A4 (en) 2022-01-19
AU2019257708A1 (en) 2020-09-03
IL277036A (en) 2020-10-29
JP2021521849A (ja) 2021-08-30
JP2026099845A (ja) 2026-06-18

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