HK1257668A1 - 短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途 - Google Patents

短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途 Download PDF

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Publication number
HK1257668A1
HK1257668A1 HK19100028.5A HK19100028A HK1257668A1 HK 1257668 A1 HK1257668 A1 HK 1257668A1 HK 19100028 A HK19100028 A HK 19100028A HK 1257668 A1 HK1257668 A1 HK 1257668A1
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cells
genetically modified
shrna734
gene
hairpin rna
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HK19100028.5A
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English (en)
Chinese (zh)
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Dong Sung An
Saki Shimizu
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The Regents Of The University Of California
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Publication of HK1257668A1 publication Critical patent/HK1257668A1/zh

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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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HK19100028.5A 2016-02-19 2017-02-17 短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途 HK1257668A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662297432P 2016-02-19 2016-02-19
US62/297,432 2016-02-19
PCT/US2017/018483 WO2017143266A1 (en) 2016-02-19 2017-02-17 Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells

Publications (1)

Publication Number Publication Date
HK1257668A1 true HK1257668A1 (zh) 2019-10-25

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HK19100028.5A HK1257668A1 (zh) 2016-02-19 2017-02-17 短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途

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US (2) US11377659B2 (https=)
EP (1) EP3416660A4 (https=)
JP (2) JP6975465B2 (https=)
KR (1) KR20180114008A (https=)
CN (1) CN108495640B (https=)
AU (1) AU2017221456B2 (https=)
HK (1) HK1257668A1 (https=)
WO (1) WO2017143266A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7410856B2 (ja) 2017-07-18 2024-01-10 シーエスエル・ベーリング・ジーン・セラピー・インコーポレイテッド ドナー改変細胞の選択のための調節可能スイッチ
CN111164211B (zh) * 2017-07-18 2024-08-02 杰特贝林基因治疗股份有限公司 用于治疗β-血红蛋白病的组合物和方法
KR20210118833A (ko) * 2018-12-23 2021-10-01 씨에스엘 베링 엘엘씨 킬 스위치를 갖는 공여 t 세포
EP3897745A1 (en) * 2018-12-23 2021-10-27 CSL Behring LLC Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
WO1997043900A1 (en) 1996-05-24 1997-11-27 The President And Fellows Of Harvard College In vivo selection
AU7605796A (en) 1996-11-04 1998-05-29 Saint Jude Children's Research Hospital (in vivo) selection of primitive hematopoietic cells
US20030032003A1 (en) 2000-02-02 2003-02-13 Schiestl Robert H. In vivo selection
WO2002040049A2 (en) 2000-11-14 2002-05-23 The General Hospital Corporation Blockade of t cell migration into epithelial gvhd target tissues
US7737124B2 (en) * 2001-09-13 2010-06-15 California Institute Of Technology Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2005056761A2 (en) * 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
EP1607482A1 (de) * 2004-06-16 2005-12-21 Gbf-Gesellschaft Für Biotechnologische Forschung Mbh RNAi-basierte Verfahren zur Selektion von transfizierten eukaryontischen Zellen
WO2008076370A2 (en) * 2006-12-18 2008-06-26 The General Hospital Corporation P21 compositions and methods for the treatment of hiv
WO2012061075A2 (en) 2010-10-25 2012-05-10 The Regents Of The University Of California Hiv resistant and functional hematopoietic stem/progenitor cells and macrophages from induced pluripotent stem cells
GB201301125D0 (en) * 2013-01-22 2013-03-06 Horizon Discovery Ltd Methods for improving the efficency of gene targeting
EP3027755B1 (en) * 2013-08-02 2019-10-09 The Regents of The University of California Engineering antiviral t cell immunity through stem cells and chimeric antigen receptors
WO2017079400A1 (en) * 2015-11-04 2017-05-11 The Trustees Of The University Of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
CN111164211B (zh) * 2017-07-18 2024-08-02 杰特贝林基因治疗股份有限公司 用于治疗β-血红蛋白病的组合物和方法

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Publication number Publication date
AU2017221456B2 (en) 2024-04-04
CN108495640A (zh) 2018-09-04
EP3416660A4 (en) 2019-11-06
EP3416660A1 (en) 2018-12-26
WO2017143266A1 (en) 2017-08-24
JP7350361B2 (ja) 2023-09-26
JP2019509034A (ja) 2019-04-04
CA3012332A1 (en) 2017-08-24
US20230076635A1 (en) 2023-03-09
CN108495640B (zh) 2022-11-04
KR20180114008A (ko) 2018-10-17
JP6975465B2 (ja) 2021-12-08
US20210310007A1 (en) 2021-10-07
JP2022033729A (ja) 2022-03-02
AU2017221456A1 (en) 2018-07-19
US11377659B2 (en) 2022-07-05

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