JP2023531729A - キルスイッチを有するドナーｔ細胞 - Google Patents

キルスイッチを有するドナーｔ細胞 Download PDF

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JP2023531729A
JP2023531729A JP2022579985A JP2022579985A JP2023531729A JP 2023531729 A JP2023531729 A JP 2023531729A JP 2022579985 A JP2022579985 A JP 2022579985A JP 2022579985 A JP2022579985 A JP 2022579985A JP 2023531729 A JP2023531729 A JP 2023531729A
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seq
sequence
guide rna
nos
rna molecule
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JP2023531729A5 (enExample
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チャオ－グアン・チェン
ワリッド・ジャン・アザール
アーサー・リアン－チー・シュー
クリストファー・ウォルター・アルマ
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シーエスエル・ベーリング・エルエルシー
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    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2510/00—Genetically modified cells

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022579985A 2020-06-26 2021-06-25 キルスイッチを有するドナーｔ細胞 Pending JP2023531729A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063044697P 2020-06-26 2020-06-26
US63/044,697 2020-06-26
PCT/US2021/039022 WO2021263070A1 (en) 2020-06-26 2021-06-25 Donor t-cells with kill switch

Publications (2)

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JP2023531729A true JP2023531729A (ja) 2023-07-25
JP2023531729A5 JP2023531729A5 (enExample) 2024-12-13

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US (1) US20230355674A1 (enExample)
EP (1) EP4172335A1 (enExample)
JP (1) JP2023531729A (enExample)
CN (1) CN115997015A (enExample)
AU (1) AU2021294317A1 (enExample)
WO (1) WO2021263070A1 (enExample)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝的状態の処置のための方法および組成物
WO2019018491A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. MODULAR SWITCH FOR SELECTING DONOR MODIFIED CELLS
JP2020508693A (ja) * 2017-03-06 2020-03-26 インスティテュート フォー ベーシック サイエンスＩｎｓｔｉｔｕｔｅ Ｆｏｒ Ｂａｓｉｃ Ｓｃｉｅｎｃｅ Ｃ２ｃ１エンドヌクレアーゼを含むゲノム編集用組成物およびこれを用いたゲノム編集方法

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5958928A (en) 1995-03-27 1999-09-28 Chugai Seiyaku Kabushiki Kaisha Pharmaceutical agents containing methotrexate derivative
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007098089A2 (en) 2006-02-17 2007-08-30 Novacea, Inc. Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
US9283194B2 (en) 2010-04-16 2016-03-15 The Regents Of The University Of California Methods for protease assisted protein delivery
US9993440B2 (en) 2011-09-02 2018-06-12 The Regents Of The University Of California Enzyme responsive nanocapsules for protein delivery
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CA2907095C (en) 2012-03-16 2020-11-03 The Regents Of The University Of California A novel rnai molecule delivery platform based on single-sirna and shrna nanocapsules
HUE054260T2 (hu) 2012-09-06 2021-08-30 Univ Chicago Antiszensz polinukleotidok exon-ugrás indukálására és eljárások disztrófiák kezelésére
JP6502261B2 (ja) 2012-12-14 2019-04-17 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア ターゲティング遺伝子治療のためのウイルスベクターナノカプセル、およびその調製
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CN107530445B (zh) 2014-11-26 2021-01-05 加利福尼亚大学董事会 用于蛋白质递送的隐形纳米胶囊
HK1247963A1 (zh) 2015-05-18 2018-10-05 Csl贝林基因治疗股份有限公司 用於hiv的治疗的基因疗法及其用途
GB2592821B (en) * 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
EP3463428A4 (en) 2016-05-24 2020-01-08 The Regents of the University of California GROWTH FACTOR NANO CAPSULES WITH TUNABLE RELEASE FOR BONE REGENERATION
US20210228630A1 (en) * 2018-05-16 2021-07-29 Research Institute At Nationwide Children's Hospital Generation of knock-out primary and expanded human nk cells using cas9 ribonucleoproteins
KR20210118833A (ko) * 2018-12-23 2021-10-01 씨에스엘 베링 엘엘씨 킬 스위치를 갖는 공여 t 세포

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521975A (ja) * 2013-05-15 2016-07-28 サンガモ バイオサイエンシーズ， インコーポレイテッド 遺伝的状態の処置のための方法および組成物
JP2020508693A (ja) * 2017-03-06 2020-03-26 インスティテュート フォー ベーシック サイエンスＩｎｓｔｉｔｕｔｅ Ｆｏｒ Ｂａｓｉｃ Ｓｃｉｅｎｃｅ Ｃ２ｃ１エンドヌクレアーゼを含むゲノム編集用組成物およびこれを用いたゲノム編集方法
WO2019018491A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. MODULAR SWITCH FOR SELECTING DONOR MODIFIED CELLS

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LIAO, S. ET AL.: "Enriching CRISPR-Cas9 targeted cells by co-targeting the HPRT gene", NUCLEIC ACIDS RESEARCH, vol. 43, no. 20, JPN6025022641, 2015, pages 134, ISSN: 0005765242 *

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CN115997015A (zh) 2023-04-21
AU2021294317A1 (en) 2023-02-23
WO2021263070A1 (en) 2021-12-30
US20230355674A1 (en) 2023-11-09
EP4172335A1 (en) 2023-05-03

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