CN111447954A - 用于恢复hbb基因功能的腺相关病毒组合物及其使用方法 - Google Patents

用于恢复hbb基因功能的腺相关病毒组合物及其使用方法 Download PDF

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Publication number
CN111447954A
CN111447954A CN201880079840.8A CN201880079840A CN111447954A CN 111447954 A CN111447954 A CN 111447954A CN 201880079840 A CN201880079840 A CN 201880079840A CN 111447954 A CN111447954 A CN 111447954A
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China
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amino acid
seq
capsid protein
aav
acid corresponding
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Pending
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CN201880079840.8A
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English (en)
Chinese (zh)
Inventor
S·查特瑞
K·K·王
M·本哈杰萨拉赫
L·J·史密斯
A·B·西摩
J·B·莱特
J·A·麦克斯维根
S·N·多里维
T·B·圣马丁
J·普劳特
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City of Hope
Cognate3 LLC
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City of Hope
Cognate3 LLC
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Publication of CN111447954A publication Critical patent/CN111447954A/zh
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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CN201880079840.8A 2017-10-18 2018-10-17 用于恢复hbb基因功能的腺相关病毒组合物及其使用方法 Pending CN111447954A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762574163P 2017-10-18 2017-10-18
US62/574,163 2017-10-18
US201862621102P 2018-01-24 2018-01-24
US62/621,102 2018-01-24
PCT/US2018/056271 WO2019079437A1 (en) 2017-10-18 2018-10-17 ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING THE FUNCTION OF THE HBB GENE AND METHODS OF USE

Publications (1)

Publication Number Publication Date
CN111447954A true CN111447954A (zh) 2020-07-24

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CN201880079840.8A Pending CN111447954A (zh) 2017-10-18 2018-10-17 用于恢复hbb基因功能的腺相关病毒组合物及其使用方法

Country Status (12)

Country Link
US (1) US12201657B2 (https=)
EP (1) EP3697450A1 (https=)
JP (1) JP2021500070A (https=)
KR (1) KR20200095462A (https=)
CN (1) CN111447954A (https=)
AU (1) AU2018352855B2 (https=)
BR (1) BR112020007765A2 (https=)
CA (1) CA3079437A1 (https=)
IL (1) IL273877A (https=)
MX (1) MX2020004092A (https=)
SG (1) SG11202002737PA (https=)
WO (1) WO2019079437A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109536494A (zh) * 2017-10-31 2019-03-29 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
EP4085143B1 (en) * 2019-12-31 2025-08-13 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
CN111363756B (zh) * 2020-04-02 2023-01-17 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种珠蛋白基因双表达慢病毒载体及其应用
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
WO2022221319A2 (en) * 2021-04-12 2022-10-20 Graphite Bio, Inc. Methods and compositions for production of genetically modified primary cells
US20250127812A1 (en) * 2021-08-10 2025-04-24 GentiBio. Inc. Compositions and methods for engineering stable tregs

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101348786A (zh) * 2007-07-20 2009-01-21 谭孟群 一种人β珠蛋白基因及其重组腺相关病毒载体
WO2016049230A1 (en) * 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016134338A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5449759A (en) * 1987-05-16 1995-09-12 Somatogen, Inc. Hemoglobins with intersubunit desulfide bonds
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE69727492T2 (de) * 1996-12-05 2004-12-16 Crucell Holland B.V. Genetische modifikation der hämatopoietischen repopulation von stammzellen in primaten
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
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
US8628966B2 (en) * 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US11242525B2 (en) * 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
CA2960209C (en) 2014-09-04 2023-08-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
US11136597B2 (en) * 2016-02-16 2021-10-05 Yale University Compositions for enhancing targeted gene editing and methods of use thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101348786A (zh) * 2007-07-20 2009-01-21 谭孟群 一种人β珠蛋白基因及其重组腺相关病毒载体
WO2016049230A1 (en) * 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016134338A1 (en) * 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
EINERHAND M. P. W. 等: "REGULATED HIGH-LEVEL HUMAN BETA-GLOBIN GENE EXPRESSION IN ERYTHROIDCELLS FOLLOWING RECOMBINANT ADENO-ASSOCIATED VIRUS-MEDIATED GENE TRANSFER" *
PONNAZHAGAN SELVARANGAN 等: "Adeno-associated virus type 2-mediated transduction of murine hematopoietic cells with long-term repopulating ability and sustained expression of a human globin gene in vivo" *

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Publication number Publication date
AU2018352855A1 (en) 2020-04-02
JP2021500070A (ja) 2021-01-07
MX2020004092A (es) 2021-01-08
IL273877A (en) 2020-05-31
KR20200095462A (ko) 2020-08-10
CA3079437A1 (en) 2019-04-25
AU2018352855B2 (en) 2022-07-14
BR112020007765A2 (pt) 2020-10-20
WO2019079437A1 (en) 2019-04-25
US20190134118A1 (en) 2019-05-09
US12201657B2 (en) 2025-01-21
EP3697450A1 (en) 2020-08-26
SG11202002737PA (en) 2020-05-28

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