JPWO2021102435A5 - - Google Patents

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Publication number
JPWO2021102435A5
JPWO2021102435A5 JP2022529542A JP2022529542A JPWO2021102435A5 JP WO2021102435 A5 JPWO2021102435 A5 JP WO2021102435A5 JP 2022529542 A JP2022529542 A JP 2022529542A JP 2022529542 A JP2022529542 A JP 2022529542A JP WO2021102435 A5 JPWO2021102435 A5 JP WO2021102435A5
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JP
Japan
Prior art keywords
raav
composition
item
delivery
ighmbp2
Prior art date
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Application number
JP2022529542A
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English (en)
Japanese (ja)
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JP2023502474A (ja
JP7684296B2 (ja
JP2023502474A5 (https=
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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 JPWO2021102435A5 publication Critical patent/JPWO2021102435A5/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
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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 (4)

Publication Number Publication Date
JP2023502474A JP2023502474A (ja) 2023-01-24
JPWO2021102435A5 true JPWO2021102435A5 (https=) 2023-11-30
JP2023502474A5 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)

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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
AU2023360998A1 (en) 2022-10-11 2025-05-22 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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2001514845A (ja) 1997-09-05 2001-09-18 ターゲティッド ジェネティクス コーポレイション 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法
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
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
EP2019588A4 (en) * 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
WO2010134939A2 (en) * 2008-12-19 2010-11-25 Zirus, Inc. Mammalian genes involved in infection
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
WO2014070949A1 (en) * 2012-11-01 2014-05-08 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
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
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
BR112019009834B1 (pt) * 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
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
TWI835747B (zh) * 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療

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