CN104540947A - 用于调节smn基因家族表达的组合物和方法 - Google Patents

用于调节smn基因家族表达的组合物和方法 Download PDF

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Publication number
CN104540947A
CN104540947A CN201380037632.9A CN201380037632A CN104540947A CN 104540947 A CN104540947 A CN 104540947A CN 201380037632 A CN201380037632 A CN 201380037632A CN 104540947 A CN104540947 A CN 104540947A
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stranded oligonucleotide
nucleotides
oligonucleotide
nucleotide
sequence
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Chinese (zh)
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阿瑟·M·克里格
罗梅什·苏布拉马尼安
詹姆斯·麦克斯维根
珍妮·T·李
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General Hospital Corp
Translate Bio Inc
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General Hospital Corp
RaNA Therapeutics Inc
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CN201380037632.9A 2012-05-16 2013-05-16 用于调节smn基因家族表达的组合物和方法 Pending CN104540947A (zh)

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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CN104540947A true CN104540947A (zh) 2015-04-22

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

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WO2021190226A1 (zh) * 2020-03-24 2021-09-30 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
WO2023020421A1 (en) * 2021-08-16 2023-02-23 Hangzhou Jiayin Biotech Ltd. Regulation of imprinting silenced genes by smn1 and snrpn expression and uses thereof
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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US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850182A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING ATP2A2 GENE EXPRESSION
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
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JP2019537427A (ja) 2016-10-27 2019-12-26 カリフォルニア インスティチュート オブ テクノロジー Ｘ染色体の再活性化のためのｈｄａｃ阻害剤組成物
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JP7689110B2 (ja) * 2019-08-15 2025-06-05 バイオジェン・エムエイ・インコーポレイテッド 脊髄性筋萎縮症のための併用療法
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