JP2022528487A - Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節 - Google Patents

Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節 Download PDF

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JP2022528487A
JP2022528487A JP2021557938A JP2021557938A JP2022528487A JP 2022528487 A JP2022528487 A JP 2022528487A JP 2021557938 A JP2021557938 A JP 2021557938A JP 2021557938 A JP2021557938 A JP 2021557938A JP 2022528487 A JP2022528487 A JP 2022528487A
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nucleotides
rna
oligonucleotide
dsrna
complementary
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JP2022528487A5 (enExample
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コボロバ，アナスタシア
ミゲル ダ クルス ゴディーニョ，ブルーノ
ダブリュー ギルバート，ジェイムズ
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University of Massachusetts Amherst
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JP2021557938A 2019-03-29 2020-03-27 Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節 Pending JP2022528487A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962826589P 2019-03-29 2019-03-29
US62/826,589 2019-03-29
US201962864789P 2019-06-21 2019-06-21
US62/864,789 2019-06-21
US201962910842P 2019-10-04 2019-10-04
US62/910,842 2019-10-04
PCT/US2020/025432 WO2020205605A2 (en) 2019-03-29 2020-03-27 OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72

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JP2022528487A true JP2022528487A (ja) 2022-06-13
JP2022528487A5 JP2022528487A5 (enExample) 2023-04-03

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US (2) US20200385737A1 (enExample)
EP (1) EP3946374A4 (enExample)
JP (1) JP2022528487A (enExample)
AU (1) AU2020252020A1 (enExample)
CA (1) CA3133784A1 (enExample)
WO (1) WO2020205605A2 (enExample)

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WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
WO2020227395A2 (en) 2019-05-06 2020-11-12 University Of Massachusetts Anti-c9orf72 oligonucleotides and related methods
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021195510A1 (en) 2020-03-27 2021-09-30 University Of Massachusetts Dual-acting sirna based modulation of c9orf72
EP4157289A4 (en) 2020-05-26 2024-06-26 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
JP2024511473A (ja) * 2021-03-24 2024-03-13 アタランタ セラピューティクス，インコーポレーテッド パターン化化学修飾を有する二本鎖ｓｉｒｎａ
EP4313070A4 (en) * 2021-03-24 2025-06-11 Atalanta Therapeutics, Inc. Microglial gene silencing using double-stranded siRNA
BR112023025224A2 (pt) * 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
US20230193281A1 (en) * 2021-11-08 2023-06-22 University Of Massachusetts Oligonucleotides for sod1 modulation
WO2023133550A2 (en) * 2022-01-10 2023-07-13 University Of Massachusetts Oligonucleotides for mecp2 modulation
EP4569116A2 (en) * 2022-08-09 2025-06-18 The University of Massachusetts Identification of allele-specific target sequences for c9orf72
CN122029280A (zh) * 2023-10-18 2026-05-12 马萨诸塞大学 具有乙二醇修饰的寡核苷酸

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WO2017109757A1 (en) * 2015-12-23 2017-06-29 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders

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WO2016201301A1 (en) * 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
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CA3064590A1 (en) * 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
TWI897902B (zh) * 2019-12-13 2025-09-21 美商阿尼拉製藥公司 人類染色體9開讀框72 (C9ORF72) iRNA劑組成物及其使用方法

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WO2015054676A2 (en) * 2013-10-11 2015-04-16 Isis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2017109757A1 (en) * 2015-12-23 2017-06-29 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
WO2019094694A1 (en) * 2017-11-10 2019-05-16 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders

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EP3946374A4 (en) 2023-07-19
US20200385737A1 (en) 2020-12-10
WO2020205605A3 (en) 2020-11-12
EP3946374A2 (en) 2022-02-09
WO2020205605A2 (en) 2020-10-08
AU2020252020A1 (en) 2021-11-11
US20240132892A1 (en) 2024-04-25
CA3133784A1 (en) 2020-10-08

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