CN110809627A - 用于xla基因疗法的优化的慢病毒载体 - Google Patents

用于xla基因疗法的优化的慢病毒载体 Download PDF

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Publication number
CN110809627A
CN110809627A CN201880043435.0A CN201880043435A CN110809627A CN 110809627 A CN110809627 A CN 110809627A CN 201880043435 A CN201880043435 A CN 201880043435A CN 110809627 A CN110809627 A CN 110809627A
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cell
seq
btk
cells
vector
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Chinese (zh)
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戴维德·J·罗林斯
凯伦·索默
斯瓦蒂·辛格
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/46Vector systems having a special element relevant for transcription elements influencing chromatin structure, e.g. scaffold/matrix attachment region, methylation free island

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CN201880043435.0A 2017-04-21 2018-04-19 用于xla基因疗法的优化的慢病毒载体 Pending CN110809627A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762488523P 2017-04-21 2017-04-21
US62/488,523 2017-04-21
PCT/US2018/028331 WO2018195297A1 (en) 2017-04-21 2018-04-19 Optimized lentiviral vector for xla gene therapy

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CN110809627A true CN110809627A (zh) 2020-02-18

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CN201880043435.0A Pending CN110809627A (zh) 2017-04-21 2018-04-19 用于xla基因疗法的优化的慢病毒载体

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Country Link
US (1) US20200325458A1 (enIt.pdf)
EP (1) EP3612238A4 (enIt.pdf)
JP (2) JP7471821B2 (enIt.pdf)
CN (1) CN110809627A (enIt.pdf)
AU (1) AU2018256412A1 (enIt.pdf)
WO (1) WO2018195297A1 (enIt.pdf)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3109924A1 (en) 2018-08-24 2020-02-27 Csl Behring Gene Therapy, Inc. Vector production in serum free media
US11464872B2 (en) 2020-12-07 2022-10-11 Noga Therapeutics Ltd. Lentiviral vectors for therapeutic expression of BTK in haematopoietic cells

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6974667B2 (en) * 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US20160004814A1 (en) * 2012-09-05 2016-01-07 University Of Washington Through Its Center For Commercialization Methods and compositions related to regulation of nucleic acids

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HANNAH KERNS等: "Lentiviral Vectors Using a Ubiquitously Acting Chromatin Opening Element and Endogenous Btk Promoter Restore B and Myeloid Cell Defects in Murine X-Linked Agammaglobulinemia", 《MOLECULAR THERAPY》 *
HANNAH M. KERNS等: "B cell–specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia", 《BLOOD》 *
SWATI SINGH等: "BTK-Promoter LV Vectors Utilizing Conserved Intron Element Mediate Functional Rescue in Murine XLA", 《MOLECULAR THERAPY》 *

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EP3612238A4 (en) 2020-12-30
JP7471821B2 (ja) 2024-04-22
JP2024084849A (ja) 2024-06-25
EP3612238A1 (en) 2020-02-26
AU2018256412A1 (en) 2019-11-07
JP2020517268A (ja) 2020-06-18
US20200325458A1 (en) 2020-10-15
WO2018195297A1 (en) 2018-10-25

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