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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Application number
JP2018529240A
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English (en)
Japanese (ja)
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JP7054675B2 (ja
JP2019500347A (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 (https=) 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)

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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 (https=)
JP (2) JP7054675B2 (https=)
CA (1) CA3005254A1 (https=)
ES (1) ES2903394T3 (https=)
WO (1) WO2017106364A2 (https=)

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GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
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
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
WO2017156242A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
KR102318434B1 (ko) 2017-08-25 2021-11-01 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
WO2019168402A1 (en) * 2018-03-02 2019-09-06 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Inhibition of polyomavirus replication
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
JP7561129B2 (ja) * 2018-12-21 2024-10-03 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２の発現を低減するための化合物及び方法
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

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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
ATE404683T1 (de) 1992-09-25 2008-08-15 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
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
ES2699827T3 (es) 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
CN103667438B (zh) * 2013-01-07 2015-04-01 赵晨 一种筛查HRDs致病突变的方法及涉及的基因芯片杂交探针设计方法
EP3041958B1 (en) 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay

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