CN107109407A - 治疗肌萎缩性侧索硬化(als)的组合物和方法 - Google Patents

治疗肌萎缩性侧索硬化(als)的组合物和方法 Download PDF

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Publication number
CN107109407A
CN107109407A CN201580072992.1A CN201580072992A CN107109407A CN 107109407 A CN107109407 A CN 107109407A CN 201580072992 A CN201580072992 A CN 201580072992A CN 107109407 A CN107109407 A CN 107109407A
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sod1
sirna
vector
aav
nucleotides
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Chinese (zh)
Inventor
D·W-Y·沙
侯金兆
M·E·农嫩马赫
周鹏程
M·霍斯巴赫
J·德克特
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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Priority to CN202210354987.1A priority Critical patent/CN114717264A/zh
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CN201580072992.1A 2014-11-14 2015-11-13 治疗肌萎缩性侧索硬化(als)的组合物和方法 Pending CN107109407A (zh)

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CN202210354987.1A CN114717264A (zh) 2014-11-14 2015-11-13 治疗肌萎缩性侧索硬化(als)的组合物和方法

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US201462079588P 2014-11-14 2014-11-14
US62/079,588 2014-11-14
US201562211992P 2015-08-31 2015-08-31
US62/211,992 2015-08-31
US201562234466P 2015-09-29 2015-09-29
US62/234,466 2015-09-29
PCT/US2015/060562 WO2016077687A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

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CN111479924A (zh) * 2017-10-16 2020-07-31 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
CN112567035A (zh) * 2018-07-02 2021-03-26 沃雅戈治疗公司 肌萎缩侧索硬化症及脊髓相关病症的治疗
CN113005123A (zh) * 2017-10-23 2021-06-22 普利维尔治疗公司 用于神经变性疾病的基因疗法
CN113365640A (zh) * 2018-12-06 2021-09-07 比奥根Ma公司 引导肌萎缩侧索硬化症的治疗性干预的神经丝蛋白
CN115397435A (zh) * 2020-04-09 2022-11-25 国立健康与医学研究所 编码人fus蛋白的核酸和在肌萎缩性侧索硬化症(als)的治疗中的用途
CN116270704A (zh) * 2023-02-24 2023-06-23 西交利物浦大学 一种抑制sod1基因表达的药物组合物及其应用

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WO2019079242A1 (en) * 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
RU2762367C1 (ru) * 2017-12-29 2021-12-20 Ньюромион Инк. Вектор на основе аденоассоциированного вируса (aav), имеющий гибридный ген hgf, введенный в него
IL279108B2 (en) * 2018-05-31 2024-10-01 Immunity Pharma Ltd Compositions and methods of using same for treating amyotrophic lateral sclerosis (als)
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UY38506A (es) * 2018-12-14 2020-06-30 Biogen Ma Inc Composiciones y metodos para tratar y prevenir la esclerosis lateral amiotrofica
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
JP2022523719A (ja) 2019-01-31 2022-04-26 オレゴン・ヘルス・アンド・サイエンス・ユニバーシティ Ａａｖキャプシドの転写依存性定方向進化を使用するための方法
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US20220389067A1 (en) * 2019-11-05 2022-12-08 The Board Of Trustees Of The Leland Stanford Junior University Methods of Treating Neurodegenerative Diseases Caused by G4C2 Expansion in C9ORF72
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
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US20230193281A1 (en) * 2021-11-08 2023-06-22 University Of Massachusetts Oligonucleotides for sod1 modulation
JP2025522433A (ja) * 2022-06-15 2025-07-15 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 スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
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