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

Publications (1)

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CN104540947A true CN104540947A (zh) 2015-04-22

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CN201380037632.9A Pending CN104540947A (zh) 2012-05-16 2013-05-16 用于调节smn基因家族表达的组合物和方法

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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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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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US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
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
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
EP2983676B1 (en) * 2013-04-12 2019-03-20 The Curators of the University of Missouri Smn2 element 1 antisense compositions and methods and uses thereof
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CN103911346B (zh) * 2014-03-27 2016-09-07 江苏雄鸣医药科技有限公司 一种用于治疗脊髓性肌萎缩症药物筛选的细胞模型
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EP4013387A4 (en) * 2019-08-15 2023-09-27 Biogen MA Inc. COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY
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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
WO2021262919A2 (en) 2020-06-26 2021-12-30 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
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KR20240126870A (ko) 2021-12-22 2024-08-21 캠프4 테라퓨틱스 코포레이션 조절 rna들을 표적으로 하는 안티센스 올리고뉴클레오티드를 사용한 유전자 전사의 조절
AU2024208843A1 (en) * 2023-01-19 2025-08-14 Research Institute At Nationwide Children's Hospital Materials and methods for modulating expression

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