JPWO2020214737A5 - - Google Patents

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Publication number
JPWO2020214737A5
JPWO2020214737A5 JP2021561810A JP2021561810A JPWO2020214737A5 JP WO2020214737 A5 JPWO2020214737 A5 JP WO2020214737A5 JP 2021561810 A JP2021561810 A JP 2021561810A JP 2021561810 A JP2021561810 A JP 2021561810A JP WO2020214737 A5 JPWO2020214737 A5 JP WO2020214737A5
Authority
JP
Japan
Prior art keywords
composition
aav
seq
polypeptide
cln6
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.)
Pending
Application number
JP2021561810A
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English (en)
Japanese (ja)
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JP2022530359A (ja
JP2022530359A5 (https=
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 JPWO2020214737A5 publication Critical patent/JPWO2020214737A5/ja
Publication of JP2022530359A5 publication Critical patent/JP2022530359A5/ja
Priority to JP2025156997A priority Critical patent/JP2025181954A/ja
Ceased legal-status Critical Current

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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 (3)

Publication Number Publication Date
JP2022530359A JP2022530359A (ja) 2022-06-29
JPWO2020214737A5 true JPWO2020214737A5 (https=) 2023-04-25
JP2022530359A5 JP2022530359A5 (https=) 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 (https=)
EP (1) EP3955969A1 (https=)
JP (2) JP2022530359A (https=)
AU (1) AU2020257208A1 (https=)
CA (1) CA3137080A1 (https=)
WO (1) WO2020214737A1 (https=)

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

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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
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
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
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
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
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
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
IN2014DN10411A (https=) * 2012-07-04 2015-08-14 Philip Morris Products Sa
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
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
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
KR20210124299A (ko) * 2019-02-04 2021-10-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cln6 폴리뉴클레오티드의 아데노-관련 바이러스 전달

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