JP2015523854A - Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 - Google Patents

Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 Download PDF

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JP2015523854A
JP2015523854A JP2015512858A JP2015512858A JP2015523854A JP 2015523854 A JP2015523854 A JP 2015523854A JP 2015512858 A JP2015512858 A JP 2015512858A JP 2015512858 A JP2015512858 A JP 2015512858A JP 2015523854 A JP2015523854 A JP 2015523854A
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stranded oligonucleotide
nucleotide
oligonucleotide
nucleotides
single stranded
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アーサー エム． クリーグ，
アーサー エム． クリーグ，
ロメシュ スブラマニアン，
ロメシュ スブラマニアン，
ジェイムズ マクスウィゲン，
ジェイムズ マクスウィゲン，
ジェニー ティー． リー，
ジェニー ティー． リー，
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ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
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JP2015512858A 2012-05-16 2013-05-16 Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 Pending JP2015523854A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261647858P 2012-05-16 2012-05-16
US61/647,858 2012-05-16
US201261719394P 2012-10-27 2012-10-27
US61/719,394 2012-10-27
US201361785529P 2013-03-14 2013-03-14
US61/785,529 2013-03-14
PCT/US2013/041440 WO2013173638A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating smn gene family expression

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JP2015523854A true JP2015523854A (ja) 2015-08-20
JP2015523854A5 JP2015523854A5 (https=) 2016-06-30

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US (1) US10059941B2 (https=)
EP (1) EP2850186B1 (https=)
JP (1) JP2015523854A (https=)
KR (1) KR20150030205A (https=)
CN (1) CN104540947A (https=)
AU (1) AU2013262649A1 (https=)
CA (1) CA2873794A1 (https=)
DK (1) DK2850186T3 (https=)
EA (1) EA201492123A1 (https=)
HK (1) HK1208700A1 (https=)
SG (1) SG11201407483YA (https=)
WO (1) WO2013173638A1 (https=)
ZA (1) ZA201409228B (https=)

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JP2018525015A (ja) * 2015-08-28 2018-09-06 サレプタ セラピューティクス，インコーポレイテッド 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12319913B2 (en) 2018-10-31 2025-06-03 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

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EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
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US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
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JP2022517008A (ja) * 2019-01-09 2022-03-03 コヨーテ バイオサイエンス ユーエスエー インコーポレイテッド 脊髄性筋萎縮症の識別のための方法およびシステム
CN111849968A (zh) 2019-04-30 2020-10-30 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用
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CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
CN113444722B (zh) * 2020-03-24 2024-09-24 中国科学院脑科学与智能技术卓越创新中心 单碱基编辑介导的剪接修复在制备治疗脊髓性肌萎缩症中的应用
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