JP2023502474A5 - - Google Patents

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Publication number
JP2023502474A5
JP2023502474A5 JP2022529542A JP2022529542A JP2023502474A5 JP 2023502474 A5 JP2023502474 A5 JP 2023502474A5 JP 2022529542 A JP2022529542 A JP 2022529542A JP 2022529542 A JP2022529542 A JP 2022529542A JP 2023502474 A5 JP2023502474 A5 JP 2023502474A5
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JP
Japan
Prior art keywords
raav
composition
item
delivery
ighmbp2
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.)
Granted
Application number
JP2022529542A
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English (en)
Japanese (ja)
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JP7684296B2 (ja
JP2023502474A (ja
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Priority claimed from PCT/US2020/061863 external-priority patent/WO2021102435A1/en
Publication of JP2023502474A publication Critical patent/JP2023502474A/ja
Publication of JP2023502474A5 publication Critical patent/JP2023502474A5/ja
Priority to JP2025081888A priority Critical patent/JP2025124694A/ja
Application granted granted Critical
Publication of JP7684296B2 publication Critical patent/JP7684296B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2022529542A 2019-11-22 2020-11-23 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 Active JP7684296B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025081888A JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962939270P 2019-11-22 2019-11-22
US62/939,270 2019-11-22
PCT/US2020/061863 WO2021102435A1 (en) 2019-11-22 2020-11-23 Materials and methods for treatment of disorders associated with the ighmbp2 gene

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025081888A Division JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

Publications (3)

Publication Number Publication Date
JP2023502474A JP2023502474A (ja) 2023-01-24
JP2023502474A5 true JP2023502474A5 (https=) 2023-11-30
JP7684296B2 JP7684296B2 (ja) 2025-05-27

Family

ID=73839115

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022529542A Active JP7684296B2 (ja) 2019-11-22 2020-11-23 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
JP2025081888A Pending JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025081888A Pending JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

Country Status (6)

Country Link
US (1) US20230211018A1 (https=)
EP (1) EP4061831A1 (https=)
JP (2) JP7684296B2 (https=)
AU (1) AU2020385387A1 (https=)
IL (1) IL293210A (https=)
WO (1) WO2021102435A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024081706A1 (en) 2022-10-11 2024-04-18 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)

Family Cites Families (28)

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PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
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US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
CN101484005A (zh) * 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
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US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9943611B2 (en) * 2012-11-01 2018-04-17 California Institute Of Technology Reversible gene expression
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
JP7106534B2 (ja) 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達
JP2020518259A (ja) * 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법

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