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
Prior art date
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)
Other versions
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 (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)

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

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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
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
CA3013797A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2018136758A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hsd17b13 variants and uses thereof
MX2019012169A (es) 2017-04-11 2019-12-11 Regeneron Pharma Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b).
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
JP7434151B2 (ja) 2017-10-11 2024-02-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｐｎｐｌａ３ ｉ１４８ｍの変異を発現している患者の肝疾患の治療におけるｈｓｄ１７ｂ１３の阻害
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
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-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
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
AU2021270720A1 (en) 2020-05-11 2022-12-08 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
CA2145535C (en) 1992-09-25 2007-07-17 Axel Kahn Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain
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
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
PT3449926T (pt) 2009-06-17 2019-11-12 Cold Spring Harbor Laboratory Composições e métodos de modulação de excisões de smn2 em um sujeito
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
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
DK3041958T3 (da) 2013-09-04 2020-03-09 Cold Spring Harbor Laboratory Reducering af nonsense-medieret mrna-degradering

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