JP2022552014A5 - - Google Patents

Info

Publication number
JP2022552014A5
JP2022552014A5 JP2022523047A JP2022523047A JP2022552014A5 JP 2022552014 A5 JP2022552014 A5 JP 2022552014A5 JP 2022523047 A JP2022523047 A JP 2022523047A JP 2022523047 A JP2022523047 A JP 2022523047A JP 2022552014 A5 JP2022552014 A5 JP 2022552014A5
Authority
JP
Japan
Prior art keywords
raav
promoter
irf2bpl
cdna
polynucleotide
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
JP2022523047A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022552014A (ja
JP7781057B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/056358 external-priority patent/WO2021077101A1/en
Publication of JP2022552014A publication Critical patent/JP2022552014A/ja
Publication of JP2022552014A5 publication Critical patent/JP2022552014A5/ja
Application granted granted Critical
Publication of JP7781057B2 publication Critical patent/JP7781057B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022523047A 2019-10-18 2020-10-19 Irf2bpl遺伝子の変異に関連する障害の治療のための材料および方法 Active JP7781057B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962923432P 2019-10-18 2019-10-18
US62/923,432 2019-10-18
PCT/US2020/056358 WO2021077101A1 (en) 2019-10-18 2020-10-19 Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene

Publications (3)

Publication Number Publication Date
JP2022552014A JP2022552014A (ja) 2022-12-14
JP2022552014A5 true JP2022552014A5 (https=) 2023-10-26
JP7781057B2 JP7781057B2 (ja) 2025-12-05

Family

ID=73402144

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022523047A Active JP7781057B2 (ja) 2019-10-18 2020-10-19 Irf2bpl遺伝子の変異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) US12606846B2 (https=)
EP (1) EP4045662A1 (https=)
JP (1) JP7781057B2 (https=)
AU (1) AU2020366242A1 (https=)
CA (1) CA3158131A1 (https=)
WO (1) WO2021077101A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115927664A (zh) * 2022-11-09 2023-04-07 苏州赛美科基因科技有限公司 一种用于快速鉴定irf2bpl突变系斑马鱼基因型的引物及其应用

Family Cites Families (24)

* 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
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
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation 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
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
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
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
JP2006296346A (ja) * 2005-04-22 2006-11-02 Kanazawa Univ 糖尿病の病態と関連して発現変動する遺伝子およびその利用
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
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
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
MX2018007330A (es) 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
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

Similar Documents

Publication Publication Date Title
JP2019511570A5 (https=)
JP7853952B2 (ja) Glut1発現のためのアデノ随伴ウイルスベクターおよびその使用
JP2022535749A5 (https=)
JP2020527333A5 (https=)
JP2020533959A5 (https=)
JP7616995B2 (ja) 神経変性疾患のための遺伝子治療
JP2021512649A5 (https=)
RU2017115477A (ru) Полинуклеотиды aadc для лечения болезни паркинсона
JP2020519629A5 (https=)
JP2023537980A5 (https=)
JP2022531809A5 (https=)
JP2021525245A5 (https=)
US20210198692A1 (en) Adeno-associated viral vector-mediated gene therapy for treating fragile x-associated disorders
JP2019537576A5 (https=)
JP2022551744A5 (https=)
WO2022017630A1 (en) GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
US20250135040A1 (en) Adeno-associated viral vector for glut1 expression and uses thereof
IL300294A (en) Gene therapy using nucleic acid constructs containing methyl CPG-binding protein 2 (MECP2) promoter sequences
JP2020509786A5 (https=)
JP2022552014A5 (https=)
JP2024543253A5 (https=)
JP2023542950A5 (https=)
JP7781057B2 (ja) Irf2bpl遺伝子の変異に関連する障害の治療のための材料および方法
JP2023502474A5 (https=)
JP2022534202A5 (https=)