JP7197466B2 - ファンコニ貧血患者に対する遺伝子療法 - Google Patents

ファンコニ貧血患者に対する遺伝子療法 Download PDF

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JP7197466B2
JP7197466B2 JP2019513876A JP2019513876A JP7197466B2 JP 7197466 B2 JP7197466 B2 JP 7197466B2 JP 2019513876 A JP2019513876 A JP 2019513876A JP 2019513876 A JP2019513876 A JP 2019513876A JP 7197466 B2 JP7197466 B2 JP 7197466B2
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cells
sequence
fanca
gene
vector
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JP2019533434A5 (enExample
JP2019533434A (ja
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フアン エイ． ビューレン
パウラ リオ
スサナ ナバーロ
フリアン セビラ
ホセ カルロス セゴビア
アフリカ ゴンサレス
ホセ アントニオ カサード
ギレルモ ゲネチェア
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Hospital Universitario Fundación Jiménez Díaz
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2730/10171—Demonstrated in vivo effect
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    • C12N2740/00011—Details
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JP2019513876A 2016-09-08 2017-09-08 ファンコニ貧血患者に対する遺伝子療法 Active JP7197466B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022117708A JP2022160505A (ja) 2016-09-08 2022-07-25 ファンコニ貧血患者に対する遺伝子療法
JP2024228258A JP2025043346A (ja) 2016-09-08 2024-12-25 ファンコニ貧血患者に対する遺伝子療法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US62/385,185 2016-09-08
US201662412028P 2016-10-24 2016-10-24
US62/412,028 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Related Child Applications (1)

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JP2022117708A Division JP2022160505A (ja) 2016-09-08 2022-07-25 ファンコニ貧血患者に対する遺伝子療法

Publications (3)

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JP2019533434A JP2019533434A (ja) 2019-11-21
JP2019533434A5 JP2019533434A5 (enExample) 2020-10-08
JP7197466B2 true JP7197466B2 (ja) 2022-12-27

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JP2019513876A Active JP7197466B2 (ja) 2016-09-08 2017-09-08 ファンコニ貧血患者に対する遺伝子療法
JP2022117708A Pending JP2022160505A (ja) 2016-09-08 2022-07-25 ファンコニ貧血患者に対する遺伝子療法
JP2024228258A Pending JP2025043346A (ja) 2016-09-08 2024-12-25 ファンコニ貧血患者に対する遺伝子療法

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JP2022117708A Pending JP2022160505A (ja) 2016-09-08 2022-07-25 ファンコニ貧血患者に対する遺伝子療法
JP2024228258A Pending JP2025043346A (ja) 2016-09-08 2024-12-25 ファンコニ貧血患者に対する遺伝子療法

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US (2) US20190203225A1 (enExample)
EP (1) EP3510162A4 (enExample)
JP (3) JP7197466B2 (enExample)
KR (2) KR20240151726A (enExample)
CN (1) CN110536966A (enExample)
AU (3) AU2017322511B2 (enExample)
CA (1) CA3035605A1 (enExample)
IL (2) IL320371A (enExample)
MX (1) MX2019002699A (enExample)
SG (1) SG11201901718VA (enExample)
WO (1) WO2018049273A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) * 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
CN110904102A (zh) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 一种用于重组蛋白质表达的启动子
CN113454235A (zh) * 2019-02-21 2021-09-28 豪夫迈·罗氏有限公司 经改进的核酸靶标富集和相关方法
KR20220012266A (ko) * 2019-05-23 2022-02-03 스페이스크래프트 세븐, 엘엘씨 영아 악성 골화석증을 위한 유전자 요법 벡터
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
AU2022264498A1 (en) * 2021-04-26 2023-09-28 Strm.Bio Incorporated Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2025137373A1 (en) * 2023-12-19 2025-06-26 Cellarity, Inc. Disease detection systems and methods

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
US20090111106A1 (en) 2000-10-06 2009-04-30 Kyri Mitrophanous Vector System
US20120071859A1 (en) 2009-04-30 2012-03-22 Morgan Richard A Inducible interleukin-12
US20120172418A1 (en) 2009-05-15 2012-07-05 Medizinische Hochscule Hannover Aslv vector system
JP2014513727A (ja) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Ｃｘｃｒ４拮抗薬の使用
WO2015056014A1 (en) 2013-10-16 2015-04-23 Ucl Business Plc Retroviral vectors
US20150203852A1 (en) 2013-11-15 2015-07-23 Northwestern University Inhibition of oxidative stress in atrial fibrillation
WO2015168547A2 (en) 2014-05-01 2015-11-05 Andre Lieber In vivo gene engineering with adenoviral vectors
WO2016118780A1 (en) 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5952190A (en) * 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
CN116622704A (zh) * 2012-07-25 2023-08-22 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
US20160194660A1 (en) * 2012-12-21 2016-07-07 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
WO2016183593A2 (en) * 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090111106A1 (en) 2000-10-06 2009-04-30 Kyri Mitrophanous Vector System
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
US20120071859A1 (en) 2009-04-30 2012-03-22 Morgan Richard A Inducible interleukin-12
US20120172418A1 (en) 2009-05-15 2012-07-05 Medizinische Hochscule Hannover Aslv vector system
JP2014513727A (ja) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Ｃｘｃｒ４拮抗薬の使用
WO2015056014A1 (en) 2013-10-16 2015-04-23 Ucl Business Plc Retroviral vectors
US20150203852A1 (en) 2013-11-15 2015-07-23 Northwestern University Inhibition of oxidative stress in atrial fibrillation
WO2015168547A2 (en) 2014-05-01 2015-11-05 Andre Lieber In vivo gene engineering with adenoviral vectors
WO2016118780A1 (en) 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Gene Ther.，2010年，17(10)，p.1244-1252
HUMAN GENE THERAPY，2010年，Vol.21，p.1-8，DOI:10.1089/hum.2009.141
Molecular Therapy，2011年，Vol.19, No.7，p.1193-1198，doi:10.1038/mt.2011.78

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