JP2022519597A5 - - Google Patents

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Publication number
JP2022519597A5
JP2022519597A5 JP2021545402A JP2021545402A JP2022519597A5 JP 2022519597 A5 JP2022519597 A5 JP 2022519597A5 JP 2021545402 A JP2021545402 A JP 2021545402A JP 2021545402 A JP2021545402 A JP 2021545402A JP 2022519597 A5 JP2022519597 A5 JP 2022519597A5
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
acid molecule
composition according
younger
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
JP2021545402A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022519597A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/016541 external-priority patent/WO2020163299A1/en
Publication of JP2022519597A publication Critical patent/JP2022519597A/ja
Publication of JP2022519597A5 publication Critical patent/JP2022519597A5/ja
Priority to JP2025023029A priority Critical patent/JP2025093922A/ja
Pending legal-status Critical Current

Links

JP2021545402A 2019-02-04 2020-02-04 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達 Pending JP2022519597A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025023029A JP2025093922A (ja) 2019-02-04 2025-02-17 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201962800915P 2019-02-04 2019-02-04
US62/800,915 2019-02-04
US201962880641P 2019-07-30 2019-07-30
US62/880,641 2019-07-30
US201962881151P 2019-07-31 2019-07-31
US62/881,151 2019-07-31
US201962912977P 2019-10-09 2019-10-09
US62/912,977 2019-10-09
US201962923125P 2019-10-18 2019-10-18
US62/923,125 2019-10-18
PCT/US2020/016541 WO2020163299A1 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of cln6 polynucleotide

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025023029A Division JP2025093922A (ja) 2019-02-04 2025-02-17 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達

Publications (2)

Publication Number Publication Date
JP2022519597A JP2022519597A (ja) 2022-03-24
JP2022519597A5 true JP2022519597A5 (https=) 2023-02-10

Family

ID=69771088

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021545402A Pending JP2022519597A (ja) 2019-02-04 2020-02-04 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達
JP2025023029A Pending JP2025093922A (ja) 2019-02-04 2025-02-17 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025023029A Pending JP2025093922A (ja) 2019-02-04 2025-02-17 Cln6ポリヌクレオチドのアデノ随伴ウイルス送達

Country Status (14)

Country Link
US (1) US20220202956A1 (https=)
EP (1) EP3921429A1 (https=)
JP (2) JP2022519597A (https=)
KR (1) KR20210124299A (https=)
CN (2) CN113574176B (https=)
AU (1) AU2020218501B2 (https=)
BR (1) BR112021015150A2 (https=)
CA (1) CA3127801A1 (https=)
CL (1) CL2021002051A1 (https=)
IL (1) IL285329A (https=)
MX (1) MX2021009404A (https=)
SG (1) SG11202107983TA (https=)
TW (1) TW202033768A (https=)
WO (1) WO2020163299A1 (https=)

Families Citing this family (4)

* 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
CA3137080A1 (en) * 2019-04-15 2020-10-22 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells
WO2026039311A1 (en) 2024-08-10 2026-02-19 Vectorgen Lentiviral vector for treating human immunodeficiency virus infection

Family Cites Families (30)

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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav 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
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
SE9403613D0 (sv) * 1994-10-21 1994-10-21 Pharmacia Ab Improvement of an expression vector for production of recombinant proteins
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
JP3765574B2 (ja) * 2001-02-22 2006-04-12 三菱化学株式会社 逆向き反復配列を含む組み換え遺伝子及びその利用
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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
US9943611B2 (en) * 2012-11-01 2018-04-17 California Institute Of Technology Reversible gene expression
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
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3411484B1 (en) * 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

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