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 (cg-RX-API-DMAC7.html
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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Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz
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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

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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

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JP2019533434A JP2019533434A (ja) 2019-11-21
JP2019533434A5 JP2019533434A5 (cg-RX-API-DMAC7.html) 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 (1) US20190203225A1 (cg-RX-API-DMAC7.html)
EP (1) EP3510162A4 (cg-RX-API-DMAC7.html)
JP (3) JP7197466B2 (cg-RX-API-DMAC7.html)
KR (2) KR102672636B1 (cg-RX-API-DMAC7.html)
CN (1) CN110536966A (cg-RX-API-DMAC7.html)
AU (3) AU2017322511B2 (cg-RX-API-DMAC7.html)
CA (1) CA3035605A1 (cg-RX-API-DMAC7.html)
IL (2) IL265196B2 (cg-RX-API-DMAC7.html)
MX (1) MX2019002699A (cg-RX-API-DMAC7.html)
SG (1) SG11201901718VA (cg-RX-API-DMAC7.html)
WO (1) WO2018049273A1 (cg-RX-API-DMAC7.html)

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KR102726267B1 (ko) 2017-10-16 2024-11-11 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터
CA3093708A1 (en) * 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for treating fanconi anemia
WO2019210325A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
SG11202100022SA (en) * 2018-07-12 2021-01-28 Rocket Pharmaceuticals Ltd 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
WO2020169666A1 (en) * 2019-02-21 2020-08-27 F. Hoffmann-La Roche Ag Improved nucleic acid target enrichment and related methods
AU2020278046A1 (en) * 2019-05-23 2022-01-27 Spacecraft Seven, Llc Gene therapy vectors for infantile malignant osteopetrosis
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
EP4192487A4 (en) 2020-08-07 2024-10-02 Spacecraft Seven, LLC GENE THERAPY FOR PLAKOPHILIN-2 (PKP2) INVOLVING AAV VECTOR
WO2022232193A1 (en) * 2021-04-26 2022-11-03 Strm.Bio Incorporated Compositions and methods related to megakaryocyte-derived extracellular vesicles for fanconi anemia
EP4499153A2 (en) * 2022-03-25 2025-02-05 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

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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
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