JPWO2020163300A5 - - Google Patents

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Publication number
JPWO2020163300A5
JPWO2020163300A5 JP2021545401A JP2021545401A JPWO2020163300A5 JP WO2020163300 A5 JPWO2020163300 A5 JP WO2020163300A5 JP 2021545401 A JP2021545401 A JP 2021545401A JP 2021545401 A JP2021545401 A JP 2021545401A JP WO2020163300 A5 JPWO2020163300 A5 JP WO2020163300A5
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JP
Japan
Prior art keywords
nucleic acid
composition
acid molecule
seq
sequence
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Application number
JP2021545401A
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English (en)
Japanese (ja)
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JP2022519596A (ja
JP7691367B2 (ja
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Priority claimed from PCT/US2020/016542 external-priority patent/WO2020163300A1/en
Publication of JP2022519596A publication Critical patent/JP2022519596A/ja
Publication of JPWO2020163300A5 publication Critical patent/JPWO2020163300A5/ja
Priority to JP2025090921A priority Critical patent/JP2025138648A/ja
Application granted granted Critical
Publication of JP7691367B2 publication Critical patent/JP7691367B2/ja
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JP2021545401A 2019-02-04 2020-02-04 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達 Active JP7691367B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025090921A JP2025138648A (ja) 2019-02-04 2025-05-30 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800911P 2019-02-04 2019-02-04
US62/800,911 2019-02-04
PCT/US2020/016542 WO2020163300A1 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of cln3 polynucleotide

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025090921A Division JP2025138648A (ja) 2019-02-04 2025-05-30 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達

Publications (3)

Publication Number Publication Date
JP2022519596A JP2022519596A (ja) 2022-03-24
JPWO2020163300A5 true JPWO2020163300A5 (enExample) 2023-02-10
JP7691367B2 JP7691367B2 (ja) 2025-06-11

Family

ID=69771089

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021545401A Active JP7691367B2 (ja) 2019-02-04 2020-02-04 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達
JP2025090921A Pending JP2025138648A (ja) 2019-02-04 2025-05-30 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達

Family Applications After (1)

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JP2025090921A Pending JP2025138648A (ja) 2019-02-04 2025-05-30 Cln3ポリヌクレオチドのアデノ随伴ウイルス送達

Country Status (16)

Country Link
US (1) US12404523B2 (enExample)
EP (1) EP3921430A1 (enExample)
JP (2) JP7691367B2 (enExample)
KR (1) KR20210124300A (enExample)
CN (1) CN113646436A (enExample)
AR (1) AR118005A1 (enExample)
AU (1) AU2020217708A1 (enExample)
BR (1) BR112021015285A2 (enExample)
CA (1) CA3129077A1 (enExample)
CL (1) CL2021002049A1 (enExample)
EA (1) EA202192160A1 (enExample)
IL (1) IL285328A (enExample)
MX (1) MX2021009401A (enExample)
SG (1) SG11202107986YA (enExample)
TW (1) TW202045530A (enExample)
WO (1) WO2020163300A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2879719B1 (en) 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
WO2022170038A1 (en) * 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
JP2025524569A (ja) * 2022-07-06 2025-07-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Cln1ポリヌクレオチドのアデノ随伴ウイルス送達

Family Cites Families (25)

* 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
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
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
CA2230758A1 (en) 1995-09-08 1997-03-13 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
CA2264483C (en) 1996-09-06 2011-03-22 James M. Wilson 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
EP1944362B1 (en) 1997-09-05 2015-10-21 Genzyme Corporation Methods for generating high titer helper-free preparations of 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
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・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 異種キャプシド中にシュードタイピングされたaav5キャプシドおよびaav5ベクターを含む組換えaavベクター
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
WO2013078316A1 (en) 2011-11-23 2013-05-30 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
EP2879719B1 (en) * 2012-08-01 2018-07-04 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
CA2967468A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
SG10202105090WA (en) * 2016-11-17 2021-06-29 Nationwide Childrens Hospital Inc Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

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