KR20210124300A - Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달 - Google Patents

Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달 Download PDF

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KR20210124300A
KR20210124300A KR1020217027483A KR20217027483A KR20210124300A KR 20210124300 A KR20210124300 A KR 20210124300A KR 1020217027483 A KR1020217027483 A KR 1020217027483A KR 20217027483 A KR20217027483 A KR 20217027483A KR 20210124300 A KR20210124300 A KR 20210124300A
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cln3
nucleic acid
seq
scaav9
raav9
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Korean (ko)
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캐스린 마이어
브라이언 케이. 카스파
케빈 파우스트
Original Assignee
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
오하이오 스테이트 이노베이션 파운데이션
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Application filed by 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈, 오하이오 스테이트 이노베이션 파운데이션 filed Critical 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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KR1020217027483A 2019-02-04 2020-02-04 Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달 Pending KR20210124300A (ko)

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

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KR20210124300A true KR20210124300A (ko) 2021-10-14

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KR1020217027483A Pending KR20210124300A (ko) 2019-02-04 2020-02-04 Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달

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US (2) US12404523B2 (https=)
EP (1) EP3921430A1 (https=)
JP (2) JP7691367B2 (https=)
KR (1) KR20210124300A (https=)
CN (1) CN113646436A (https=)
AR (1) AR118005A1 (https=)
AU (1) AU2020217708B2 (https=)
BR (1) BR112021015285A2 (https=)
CA (1) CA3129077A1 (https=)
CL (1) CL2021002049A1 (https=)
EA (1) EA202192160A1 (https=)
IL (1) IL285328A (https=)
MX (1) MX2021009401A (https=)
SG (1) SG11202107986YA (https=)
TW (1) TW202045530A (https=)
WO (1) WO2020163300A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013296425B2 (en) 2012-08-01 2018-06-07 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
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
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
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
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

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TW202045530A (zh) 2020-12-16
CN113646436A (zh) 2021-11-12
EA202192160A1 (ru) 2021-11-17
US12404523B2 (en) 2025-09-02
AR118005A1 (es) 2021-09-08
JP2025138648A (ja) 2025-09-25
WO2020163300A1 (en) 2020-08-13
AU2020217708B2 (en) 2026-01-22
SG11202107986YA (en) 2021-08-30
US20260062713A1 (en) 2026-03-05
EP3921430A1 (en) 2021-12-15
MX2021009401A (es) 2021-11-12
CA3129077A1 (en) 2020-08-13
BR112021015285A2 (pt) 2021-10-05
JP7691367B2 (ja) 2025-06-11
US20220127641A1 (en) 2022-04-28
IL285328A (en) 2021-09-30
AU2020217708A1 (en) 2021-08-19
CL2021002049A1 (es) 2022-04-01
WO2020163300A9 (en) 2021-04-15
JP2022519596A (ja) 2022-03-24

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