JP2022530359A5 - - Google Patents

Info

Publication number
JP2022530359A5
JP2022530359A5 JP2021561810A JP2021561810A JP2022530359A5 JP 2022530359 A5 JP2022530359 A5 JP 2022530359A5 JP 2021561810 A JP2021561810 A JP 2021561810A JP 2021561810 A JP2021561810 A JP 2021561810A JP 2022530359 A5 JP2022530359 A5 JP 2022530359A5
Authority
JP
Japan
Prior art keywords
aav
seq
polypeptide
cln6
item
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.)
Ceased
Application number
JP2021561810A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022530359A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/028366 external-priority patent/WO2020214737A1/en
Publication of JP2022530359A publication Critical patent/JP2022530359A/ja
Publication of JP2022530359A5 publication Critical patent/JP2022530359A5/ja
Priority to JP2025156997A priority Critical patent/JP2025181954A/ja
Ceased legal-status Critical Current

Links

JP2021561810A 2019-04-15 2020-04-15 バッテン病の視覚効果を治療または予防するための遺伝子療法 Ceased JP2022530359A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025156997A JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962834340P 2019-04-15 2019-04-15
US62/834,340 2019-04-15
PCT/US2020/028366 WO2020214737A1 (en) 2019-04-15 2020-04-15 Gene therapy for treating or preventing visual effects in batten disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025156997A Division JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

Publications (2)

Publication Number Publication Date
JP2022530359A JP2022530359A (ja) 2022-06-29
JP2022530359A5 true JP2022530359A5 (enExample) 2023-04-25

Family

ID=70739153

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021561810A Ceased JP2022530359A (ja) 2019-04-15 2020-04-15 バッテン病の視覚効果を治療または予防するための遺伝子療法
JP2025156997A Pending JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025156997A Pending JP2025181954A (ja) 2019-04-15 2025-09-22 バッテン病の視覚効果を治療または予防するための遺伝子療法

Country Status (6)

Country Link
US (1) US20220193259A1 (enExample)
EP (1) EP3955969A1 (enExample)
JP (2) JP2022530359A (enExample)
AU (1) AU2020257208A1 (enExample)
CA (1) CA3137080A1 (enExample)
WO (1) WO2020214737A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119213133B (zh) * 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
CN120842306B (zh) * 2025-07-17 2026-01-06 渤海大学 一种仿刺参精抗氧化肽及其制备方法和应用

Family Cites Families (26)

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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
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
KR102220688B1 (ko) * 2012-07-04 2021-03-03 필립모리스 프로덕츠 에스.에이. 개선된 결합제를 갖는 가연성 열원
AU2013296425B2 (en) * 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
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
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
JP2022519597A (ja) * 2019-02-04 2022-03-24 リサーチ・インスティチュート・アット・ネーションワイド・チルドレンズ・ホスピタル Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達

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