JP2016528887A5 - - Google Patents

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Publication number
JP2016528887A5
JP2016528887A5 JP2016524365A JP2016524365A JP2016528887A5 JP 2016528887 A5 JP2016528887 A5 JP 2016528887A5 JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016528887 A5 JP2016528887 A5 JP 2016528887A5
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JP
Japan
Prior art keywords
nucleic acid
oligonucleotide chain
nucleotides
antitrypsin
target sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2016524365A
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English (en)
Japanese (ja)
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JP2016528887A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2014/045365 external-priority patent/WO2015003113A2/en
Publication of JP2016528887A publication Critical patent/JP2016528887A/ja
Publication of JP2016528887A5 publication Critical patent/JP2016528887A5/ja
Pending legal-status Critical Current

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JP2016524365A 2013-07-03 2014-07-03 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物 Pending JP2016528887A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US61/842,551 2013-07-03
US201361891548P 2013-10-16 2013-10-16
US61/891,548 2013-10-16
PCT/US2014/045365 WO2015003113A2 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020094030A Division JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物

Publications (2)

Publication Number Publication Date
JP2016528887A JP2016528887A (ja) 2016-09-23
JP2016528887A5 true JP2016528887A5 (enExample) 2017-08-10

Family

ID=52133228

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2016524365A Pending JP2016528887A (ja) 2013-07-03 2014-07-03 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物
JP2020094030A Withdrawn JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物
JP2022019996A Withdrawn JP2022078069A (ja) 2013-07-03 2022-02-10 二本鎖RNAによるα-1アンチトリプシンの特異的阻害のための方法及び組成物
JP2024045063A Withdrawn JP2024075702A (ja) 2013-07-03 2024-03-21 二本鎖RNAによるα-1アンチトリプシンの特異的阻害のための方法及び組成物

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2020094030A Withdrawn JP2020188771A (ja) 2013-07-03 2020-05-29 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物
JP2022019996A Withdrawn JP2022078069A (ja) 2013-07-03 2022-02-10 二本鎖RNAによるα-1アンチトリプシンの特異的阻害のための方法及び組成物
JP2024045063A Withdrawn JP2024075702A (ja) 2013-07-03 2024-03-21 二本鎖RNAによるα-1アンチトリプシンの特異的阻害のための方法及び組成物

Country Status (11)

Country Link
US (11) US9458457B2 (enExample)
EP (3) EP3017047A4 (enExample)
JP (4) JP2016528887A (enExample)
AU (2) AU2014284298B2 (enExample)
CA (1) CA2916499A1 (enExample)
DK (1) DK3444350T3 (enExample)
ES (1) ES2905257T3 (enExample)
HU (1) HUE058794T2 (enExample)
PL (1) PL3444350T3 (enExample)
PT (1) PT3444350T (enExample)
WO (1) WO2015003113A2 (enExample)

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EP3017047A4 (en) 2013-07-03 2017-06-14 Dicerna Pharmaceuticals Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
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SE1750774A1 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
CN111971051A (zh) * 2018-04-13 2020-11-20 迪克纳制药公司 用增加tm的核苷酸修饰的双链核酸抑制剂分子
EP3790964A4 (en) * 2018-05-11 2022-06-08 Beam Therapeutics, Inc. METHODS FOR REMOVAL OF PATHOGENIC MUTATIONS USING PROGRAMMABLE BASE EDITOR SYSTEMS
EP3980540A4 (en) 2019-06-06 2023-11-01 Arrowhead Pharmaceuticals, Inc. METHOD FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD)
JP7846095B2 (ja) * 2020-09-15 2026-04-14 アローヘッド ファーマシューティカルズ インコーポレイテッド Z-aatタンパク質のレベルを低下させる方法
TW202308660A (zh) * 2021-08-25 2023-03-01 美商戴瑟納製藥股份有限公司 用於抑制α-1抗胰蛋白酶表現之組合物及方法
JP2024533312A (ja) * 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー Serpina調節組成物及び方法
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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