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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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 Active JP7049248B2 (ja)

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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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CN113195719A (zh) * 2018-09-26 2021-07-30 卡斯西部储备大学 用于增加蛋白质表达和/或治疗单倍剂量不足症的方法及组合物
US20220049250A1 (en) * 2018-12-04 2022-02-17 Stichting Katholieke Universiteit Antisense Oligonucleotides Rescue Aberrant Splicing of ABCA4
AU2020210924A1 (en) * 2019-01-23 2021-09-16 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting SCN2A retained introns
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US11618900B2 (en) 2019-11-01 2023-04-04 The Johns Hopkins University Modulating SYNGAP
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CN115516094A (zh) * 2020-02-28 2022-12-23 芝加哥大学 包含反式作用翻译活化剂的方法和组合物
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