JPWO2022221574A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2022221574A5
JPWO2022221574A5 JP2023562865A JP2023562865A JPWO2022221574A5 JP WO2022221574 A5 JPWO2022221574 A5 JP WO2022221574A5 JP 2023562865 A JP2023562865 A JP 2023562865A JP 2023562865 A JP2023562865 A JP 2023562865A JP WO2022221574 A5 JPWO2022221574 A5 JP WO2022221574A5
Authority
JP
Japan
Prior art keywords
aav
composition
viral vector
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
JP2024514160A5 (https=
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
Publication of JPWO2022221574A5 publication Critical patent/JPWO2022221574A5/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 2021-04-15
US17/231,963 US12275941B2 (en) 2021-04-15 2021-04-15 Products and methods for inhibition of expression of dynamin-1 variants
PCT/US2022/024885 WO2022221574A2 (en) 2021-04-15 2022-04-14 Products and methods for inhibition of expression of dynamin-1 variants

Publications (3)

Publication Number Publication Date
JP2024514160A JP2024514160A (ja) 2024-03-28
JP2024514160A5 JP2024514160A5 (https=) 2025-04-04
JPWO2022221574A5 true JPWO2022221574A5 (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
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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
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
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
AU2002248297A1 (en) 2001-01-05 2002-07-16 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
US20110081362A1 (en) * 2008-01-31 2011-04-07 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
BR112018070249A2 (pt) * 2016-04-02 2019-01-29 Research Institute At Nationwide Children's Hospital sistema promotor de u6 modificada para expressão específica de tecido
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
WO2018200674A1 (en) 2017-04-26 2018-11-01 Mitobridge, Inc. Dynamin-1-like protein inhibitors
BR112020014425A2 (pt) 2018-01-15 2020-12-29 Ionis Pharmaceuticals, Inc. Moduladores de expressão de dnm2

Similar Documents

Publication Publication Date Title
JP7395550B2 (ja) 新規アデノ随伴ウイルスキャプシドタンパク質
US11939597B2 (en) Restrictive inverted terminal repeats for viral vectors
JP2020019772A5 (https=)
CA2562788A1 (en) Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
JP2020522269A5 (https=)
JP2025162557A5 (https=)
JP2021534755A5 (https=)
JP2007507223A5 (https=)
JP2021522842A (ja) 肝臓を標的とするアデノ随伴ウイルスベクター
IL320714A (en) Gene therapy for limb-girdle muscular dystrophy type 2c
JP2017510298A5 (https=)
JP2021534794A5 (https=)
JP2020519294A5 (https=)
CN120548368A (zh) 用于正确包装重复元件的腺相关病毒载体
JPWO2022119826A5 (https=)
JPWO2022221574A5 (https=)
IL320715A (en) Gene therapy with dual dysperlin vectors
JPWO2020041634A5 (https=)
JPWO2020047268A5 (https=)
JPWO2021222168A5 (https=)
JP2020533313A5 (https=)
JPWO2020219766A5 (https=)
RU2025127724A (ru) Молекула нуклеиновой кислоты, содержащая асимметрично модифицированный ITR для повышения уровня экспрессии встроенного гена, и ее применение
HK40115114A (zh) 针对鞘内给药优化的导入肝细胞生长因子基因的aav载体
JPWO2023060215A5 (https=)