JP2023081369A5 - - Google Patents

Info

Publication number
JP2023081369A5
JP2023081369A5 JP2022191138A JP2022191138A JP2023081369A5 JP 2023081369 A5 JP2023081369 A5 JP 2023081369A5 JP 2022191138 A JP2022191138 A JP 2022191138A JP 2022191138 A JP2022191138 A JP 2022191138A JP 2023081369 A5 JP2023081369 A5 JP 2023081369A5
Authority
JP
Japan
Prior art keywords
recombinant aav
cell
sequence
complementary
subject
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
JP2022191138A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023081369A (ja
Filing date
Publication date
Application filed filed Critical
Publication of JP2023081369A publication Critical patent/JP2023081369A/ja
Publication of JP2023081369A5 publication Critical patent/JP2023081369A5/ja
Pending legal-status Critical Current

Links

JP2022191138A 2021-11-30 2022-11-30 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 Pending JP2023081369A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163284418P 2021-11-30 2021-11-30
US63/284,418 2021-11-30

Publications (2)

Publication Number Publication Date
JP2023081369A JP2023081369A (ja) 2023-06-09
JP2023081369A5 true JP2023081369A5 (enExample) 2025-10-27

Family

ID=85278650

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022191138A Pending JP2023081369A (ja) 2021-11-30 2022-11-30 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用

Country Status (3)

Country Link
US (1) US20230279431A1 (enExample)
EP (1) EP4186919A1 (enExample)
JP (1) JP2023081369A (enExample)

Family Cites Families (24)

* 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
EP0728214B1 (en) 1993-11-09 2004-07-28 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
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
CA2625279A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
ES2317646T3 (es) 1995-09-08 2009-04-16 Genzyme Corporation Vectores aav mejorados para terapia genica.
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
CN1233291A (zh) 1996-09-06 1999-10-27 宾西法尼亚大学托管会 重组腺伴随病毒定向基因治疗的方法
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
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
AU2001255575B2 (en) 2000-04-28 2006-08-31 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
PT2359869T (pt) 2001-12-17 2019-04-16 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP3290055B1 (en) 2011-07-25 2024-08-28 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
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
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
AU2019216257B2 (en) * 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C

Similar Documents

Publication Publication Date Title
US12383587B2 (en) AAV triple-plasmid system
JP7493566B2 (ja) 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
IL275880B2 (en) Gene therapy for brachioradial muscular dystrophy type 2C
JP7646362B2 (ja) 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー
RU2018116076A (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
JP2017509632A5 (enExample)
JP2019513399A5 (enExample)
JP2017510298A5 (enExample)
HRP20192141T1 (hr) Genska terapija za retinitis pigmentosa
US20240318199A1 (en) Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders
JP2018515102A (ja) スペーサーを含む核酸分子およびその使用の方法
JPWO2019152474A5 (enExample)
JPWO2019204593A5 (enExample)
CA3228365A1 (en) Treatment of muscular dystrophy
JP2023059858A5 (enExample)
JP2023081369A5 (enExample)
JPWO2022028472A5 (enExample)
JPWO2021221995A5 (enExample)
Ikezu The Use of Viral Vectors to Enhance Cognition
Xiao Virus-based vectors for gene expression in mammalian cells: adeno-associated virus
JP2023081369A (ja) 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
RU2023121263A (ru) Вектор на основе рнк-содержащего аденоассоциированного вируса (raav) и пути его применения
JPWO2021050991A5 (enExample)
RU2024133098A (ru) Направленная генная терапия для миотонической дистрофии dm-1
NZ747137B2 (en) Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy