JP2022548800A - フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 - Google Patents

フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 Download PDF

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JP2022548800A
JP2022548800A JP2022543368A JP2022543368A JP2022548800A JP 2022548800 A JP2022548800 A JP 2022548800A JP 2022543368 A JP2022543368 A JP 2022543368A JP 2022543368 A JP2022543368 A JP 2022543368A JP 2022548800 A JP2022548800 A JP 2022548800A
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nucleic acid
fxn
expression
gene
nanoparticle
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セリーナ ルナルディ，
アダム ウォルター シャイデッガー，
ジェシー ジェローム スミス，
ジェレミア デイル ファレリ，
ジョディ ミシェル ケネディ，
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オメガ セラピューティクス， インコーポレイテッド
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JP2022543368A 2019-09-23 2020-09-23 フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 Pending JP2022548800A (ja)

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JP2025135712A JP2025169372A (ja) 2019-09-23 2025-08-18 フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物

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US201962904391P 2019-09-23 2019-09-23
US62/904,391 2019-09-23
PCT/US2020/052101 WO2021061698A1 (en) 2019-09-23 2020-09-23 Methods and compositions for modulating frataxin expression and treating friedrich's ataxia

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JP2022548800A5 JP2022548800A5 (https=) 2023-10-02

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JP2025135712A Pending JP2025169372A (ja) 2019-09-23 2025-08-18 フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物

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US (1) US20220348893A1 (https=)
EP (1) EP4034660A4 (https=)
JP (2) JP2022548800A (https=)
CN (1) CN114787353A (https=)
AU (1) AU2020352984A1 (https=)
CA (1) CA3155417A1 (https=)
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US20190255106A1 (en) 2016-09-07 2019-08-22 Flagship Pioneering Inc. Methods and compositions for modulating gene expression
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
WO2023275892A1 (en) * 2021-06-29 2023-01-05 Council Of Scientific & Industrial Research Engineered fncas9 and uses thereof
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods

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JP2014534240A (ja) * 2011-11-18 2014-12-18 ユニヴェルシテ ラヴァルＵｎｉｖｅｒｓｉｔｅ Ｌａｖａｌ フラタキシンレベルを増加させる方法および生成物ならびにその使用
JP2016517549A (ja) * 2013-02-07 2016-06-16 ザ ジェネラル ホスピタル コーポレイション Ｔａｌｅ転写活性化因子
JP2017514513A (ja) * 2014-03-20 2017-06-08 ユニベルシテ ラバル フラタキシンレベルを増加させるためのｃｒｉｓｐｒ系の方法及び生成物、ならびにそれらの使用

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US6150091A (en) * 1996-03-06 2000-11-21 Baylor College Of Medicine Direct molecular diagnosis of Friedreich ataxia
KR102043422B1 (ko) * 2011-06-09 2019-11-11 큐알엔에이, 인크. 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료
CN109476716A (zh) * 2016-03-23 2019-03-15 加利福尼亚大学董事会 治疗线粒体障碍的方法
EP4484443A3 (en) * 2016-06-29 2025-03-26 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
US11866469B2 (en) * 2019-02-06 2024-01-09 Klogenix Llc DNA binding proteins and uses thereof

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2014534240A (ja) * 2011-11-18 2014-12-18 ユニヴェルシテ ラヴァルＵｎｉｖｅｒｓｉｔｅ Ｌａｖａｌ フラタキシンレベルを増加させる方法および生成物ならびにその使用
JP2016517549A (ja) * 2013-02-07 2016-06-16 ザ ジェネラル ホスピタル コーポレイション Ｔａｌｅ転写活性化因子
JP2017514513A (ja) * 2014-03-20 2017-06-08 ユニベルシテ ラバル フラタキシンレベルを増加させるためのｃｒｉｓｐｒ系の方法及び生成物、ならびにそれらの使用

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AU2020352984A1 (en) 2022-03-31
CN114787353A (zh) 2022-07-22
WO2021061698A1 (en) 2021-04-01
EP4034660A4 (en) 2023-10-11
JP2025169372A (ja) 2025-11-12
EP4034660A1 (en) 2022-08-03
US20220348893A1 (en) 2022-11-03
CA3155417A1 (en) 2021-04-01

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