JP2019500347A5 - - Google Patents

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Publication number
JP2019500347A5
JP2019500347A5 JP2018529240A JP2018529240A JP2019500347A5 JP 2019500347 A5 JP2019500347 A5 JP 2019500347A5 JP 2018529240 A JP2018529240 A JP 2018529240A JP 2018529240 A JP2018529240 A JP 2018529240A JP 2019500347 A5 JP2019500347 A5 JP 2019500347A5
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JP
Japan
Prior art keywords
protein
mrna
prpf8
pharmaceutical composition
nucleobases
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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Application number
JP2018529240A
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English (en)
Japanese (ja)
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JP2019500347A (ja
JP7054675B2 (ja
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Priority claimed from PCT/US2016/066684 external-priority patent/WO2017106364A2/en
Publication of JP2019500347A publication Critical patent/JP2019500347A/ja
Publication of JP2019500347A5 publication Critical patent/JP2019500347A5/ja
Priority to JP2022062557A priority Critical patent/JP2022088621A/ja
Application granted granted Critical
Publication of JP7054675B2 publication Critical patent/JP7054675B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018529240A 2015-12-14 2016-12-14 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 Expired - Fee Related JP7054675B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022062557A JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267261P 2015-12-14 2015-12-14
US62/267,261 2015-12-14
PCT/US2016/066684 WO2017106364A2 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022062557A Division JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Publications (3)

Publication Number Publication Date
JP2019500347A JP2019500347A (ja) 2019-01-10
JP2019500347A5 true JP2019500347A5 (enExample) 2020-01-30
JP7054675B2 JP7054675B2 (ja) 2022-04-14

Family

ID=59057547

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529240A Expired - Fee Related JP7054675B2 (ja) 2015-12-14 2016-12-14 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法
JP2022062557A Pending JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022062557A Pending JP2022088621A (ja) 2015-12-14 2022-04-04 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法

Country Status (5)

Country Link
EP (1) EP3390642B1 (enExample)
JP (2) JP7054675B2 (enExample)
CA (1) CA3005254A1 (enExample)
ES (1) ES2903394T3 (enExample)
WO (1) WO2017106364A2 (enExample)

Families Citing this family (16)

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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
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
AU2017229778A1 (en) 2016-03-09 2018-08-16 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
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
CN112218949B (zh) * 2018-03-02 2025-02-11 莱顿大学医学中心附属莱顿教学医院 多瘤病毒复制的抑制
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
EP3897837A4 (en) * 2018-12-21 2023-08-16 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING PMP22 EXPRESSION
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
US6294520B1 (en) 1989-03-27 2001-09-25 Albert T. Naito Material for passage through the blood-brain barrier
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
DE69334238D1 (de) 1992-09-25 2008-09-25 Aventis Pharma Sa Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (fr) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
KR20220084437A (ko) 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
CN104004826B (zh) * 2013-01-07 2016-03-02 赵晨 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用
WO2015035091A1 (en) 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

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