JP7049248B2 - 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー - Google Patents

常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー Download PDF

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JP7049248B2
JP7049248B2 JP2018529250A JP2018529250A JP7049248B2 JP 7049248 B2 JP7049248 B2 JP 7049248B2 JP 2018529250 A JP2018529250 A JP 2018529250A JP 2018529250 A JP2018529250 A JP 2018529250A JP 7049248 B2 JP7049248 B2 JP 7049248B2
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aso
intron
syngap1
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アズナレズ，イザベル
エム． ナッシュ，ヒュー
クライナー，エイドリアン
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コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス，インク．
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JP2018529250A 2015-12-14 2016-12-14 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー Expired - Fee Related JP7049248B2 (ja)

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JP2022013860A JP2022062140A (ja) 2015-12-14 2022-02-01 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー

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US201562267251P 2015-12-14 2015-12-14
US62/267,251 2015-12-14
PCT/US2016/066708 WO2017106377A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome

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JP2022013860A Pending JP2022062140A (ja) 2015-12-14 2022-02-01 常染色体優性精神遅滞－５とドラベ症候群の処置のためのアンチセンスオリゴマー

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WO2017106377A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
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CN120290560A (zh) * 2017-10-23 2025-07-11 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
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CN113329739B (zh) * 2018-08-20 2024-06-25 罗贡股份有限公司 用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸
KR102814622B1 (ko) * 2018-09-26 2025-05-29 케이스 웨스턴 리저브 유니버시티 단백질 발현 증가용 및/또는 반가불충분성 장애 치료용 방법 및 조성물
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EP4110918A4 (en) * 2020-02-28 2024-04-03 The University of Chicago METHODS AND COMPOSITIONS COMPRISING TRANS-ACTING TRANSLATION ACTIVATORS
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
KR20230009965A (ko) * 2020-05-11 2023-01-17 더 플로레이 인스티튜트 오브 뉴로사이언스 앤드 멘탈 헬스 Syngap1에서 기능-상실 돌연변이와 연관된 장애를 치료하기 위한 조성물 및 방법
CN114480651B (zh) * 2022-02-14 2023-10-20 天津市泌尿外科研究所 Pcat1的反义寡核苷酸及其在制备抑制前列腺癌核酸药物中的应用
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WO2024145496A2 (en) * 2022-12-28 2024-07-04 The University Of Chicago Splice-switching oligonucleotides for treating syngap1-associated disorders
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