JP2024514160A5 - - Google Patents

Info

Publication number
JP2024514160A5
JP2024514160A5 JP2023562865A JP2023562865A JP2024514160A5 JP 2024514160 A5 JP2024514160 A5 JP 2024514160A5 JP 2023562865 A JP2023562865 A JP 2023562865A JP 2023562865 A JP2023562865 A JP 2023562865A JP 2024514160 A5 JP2024514160 A5 JP 2024514160A5
Authority
JP
Japan
Prior art keywords
aav
viral vector
composition according
dnm1
nucleic acid
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
JP2023562865A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024514160A (ja
Filing date
Publication date
Priority claimed from US17/231,963 external-priority patent/US12275941B2/en
Application filed filed Critical
Publication of JP2024514160A publication Critical patent/JP2024514160A/ja
Publication of JP2024514160A5 publication Critical patent/JP2024514160A5/ja
Pending legal-status Critical Current

Links

JP2023562865A 2021-04-15 2022-04-14 ダイナミン1バリアントの発現の阻害のための産物及び方法 Pending JP2024514160A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17/231,963 US12275941B2 (en) 2021-04-15 2021-04-15 Products and methods for inhibition of expression of dynamin-1 variants
US17/231,963 2021-04-15
PCT/US2022/024885 WO2022221574A2 (en) 2021-04-15 2022-04-14 Products and methods for inhibition of expression of dynamin-1 variants

Publications (2)

Publication Number Publication Date
JP2024514160A JP2024514160A (ja) 2024-03-28
JP2024514160A5 true JP2024514160A5 (https=) 2025-04-04

Family

ID=83602203

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023562865A Pending JP2024514160A (ja) 2021-04-15 2022-04-14 ダイナミン1バリアントの発現の阻害のための産物及び方法

Country Status (4)

Country Link
US (2) US12275941B2 (https=)
EP (1) EP4322964A4 (https=)
JP (1) JP2024514160A (https=)
WO (1) WO2022221574A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025226343A1 (en) 2024-04-26 2025-10-30 Research Institute At Nationwide Children's Hospital Products and methods to inhibit expression of dynamin-1 variants and replace dynamin-1

Family Cites Families (26)

* 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
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US20070225363A1 (en) * 2003-11-21 2007-09-27 The University Of Newcastle Research Associates Ltd. Methods and Agents for Inhibiting Dynamin-Dependent Endocytosis
WO2007056435A2 (en) 2005-11-08 2007-05-18 The General Hospital Corporation Dynamin mediated diseases and associated methods and products
US20080306007A1 (en) * 2007-04-30 2008-12-11 Newcastle Innovation Limited Agents for prophylaxis or treatment of neurological related diseases and conditions
WO2009099991A2 (en) * 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CA3019832C (en) * 2016-04-02 2023-05-09 Research Institute At Nationwide Children's Hospital Modified u6 promoter system for tissue specific expression
US10947540B2 (en) 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
EP3610016A1 (en) 2017-04-10 2020-02-19 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
US11168080B2 (en) 2017-04-26 2021-11-09 Mitobridge, Inc. Dynamin-1-like protein inhibitors
AU2019206731A1 (en) 2018-01-15 2020-07-30 Ionis Pharmaceuticals, Inc. Modulators of DNM2 expression

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