JP7051683B2 - 結節性硬化症の処置のためのアンチセンスオリゴマー - Google Patents

結節性硬化症の処置のためのアンチセンスオリゴマー Download PDF

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JP7051683B2
JP7051683B2 JP2018529258A JP2018529258A JP7051683B2 JP 7051683 B2 JP7051683 B2 JP 7051683B2 JP 2018529258 A JP2018529258 A JP 2018529258A JP 2018529258 A JP2018529258 A JP 2018529258A JP 7051683 B2 JP7051683 B2 JP 7051683B2
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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JP2018529258A 2015-12-14 2016-12-14 結節性硬化症の処置のためのアンチセンスオリゴマー Expired - Fee Related JP7051683B2 (ja)

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JP2022053948A JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症の処置のためのアンチセンスオリゴマー

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US201562267212P 2015-12-14 2015-12-14
US62/267,212 2015-12-14
PCT/US2016/066705 WO2017106375A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of tuberous sclerosis complex

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JP2019500350A JP2019500350A (ja) 2019-01-10
JP2019500350A5 JP2019500350A5 (enExample) 2020-01-30
JP7051683B2 true JP7051683B2 (ja) 2022-04-11

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JP2022053948A Pending JP2022088533A (ja) 2015-12-14 2022-03-29 結節性硬化症の処置のためのアンチセンスオリゴマー

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GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
CA3059348A1 (en) 2017-04-11 2018-10-18 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
KR20260009385A (ko) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
MY207352A (en) 2018-03-21 2025-02-21 Regeneron Pharma 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
EP4560019A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions
EP4560020A1 (en) * 2023-11-22 2025-05-28 Sylentis S.A.U. Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions

Citations (1)

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Publication number Priority date Publication date Assignee Title
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex

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US20110150897A1 (en) * 2006-10-11 2011-06-23 Meyer Thomas F Influenza targets
EP3135772B1 (en) * 2009-09-08 2019-10-23 Laboratory Corporation of America Holdings Method for diagnosing autism spectrum disorders
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
WO2017106382A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex

Non-Patent Citations (3)

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CANCER RES.，2002年，Vol.62，p.1503-1509
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EP3390635A1 (en) 2018-10-24
CA3005131A1 (en) 2017-06-22
WO2017106375A1 (en) 2017-06-22
JP2019500350A (ja) 2019-01-10
JP2022088533A (ja) 2022-06-14
EP3390635A4 (en) 2019-05-01

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