JPWO2020106916A5 - - Google Patents

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Publication number
JPWO2020106916A5
JPWO2020106916A5 JP2021529002A JP2021529002A JPWO2020106916A5 JP WO2020106916 A5 JPWO2020106916 A5 JP WO2020106916A5 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP WO2020106916 A5 JPWO2020106916 A5 JP WO2020106916A5
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JP
Japan
Prior art keywords
sequence
nucleic acid
seq
nos
aav vector
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Application number
JP2021529002A
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English (en)
Japanese (ja)
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JP7704998B2 (ja
JP2022508182A (ja
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Priority claimed from PCT/US2019/062531 external-priority patent/WO2020106916A1/en
Publication of JP2022508182A publication Critical patent/JP2022508182A/ja
Publication of JPWO2020106916A5 publication Critical patent/JPWO2020106916A5/ja
Priority to JP2025034621A priority Critical patent/JP2025102783A/ja
Application granted granted Critical
Publication of JP7704998B2 publication Critical patent/JP7704998B2/ja
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JP2021529002A 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸 Active JP7704998B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025034621A JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
US62/770,202 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025034621A Division JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Publications (3)

Publication Number Publication Date
JP2022508182A JP2022508182A (ja) 2022-01-19
JPWO2020106916A5 true JPWO2020106916A5 (enExample) 2022-11-30
JP7704998B2 JP7704998B2 (ja) 2025-07-09

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ID=70773595

Family Applications (2)

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JP2021529002A Active JP7704998B2 (ja) 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸
JP2025034621A Pending JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Family Applications After (1)

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JP2025034621A Pending JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Country Status (20)

Country Link
US (1) US20210324418A1 (enExample)
EP (1) EP3883954A4 (enExample)
JP (2) JP7704998B2 (enExample)
KR (1) KR20210103469A (enExample)
CN (1) CN113302201A (enExample)
AR (1) AR117145A1 (enExample)
AU (1) AU2019385506A1 (enExample)
BR (1) BR112021009733A2 (enExample)
CA (1) CA3120289A1 (enExample)
CL (1) CL2021001327A1 (enExample)
CO (1) CO2021008120A2 (enExample)
EA (1) EA202191418A1 (enExample)
EC (1) ECSP21044840A (enExample)
IL (1) IL283344A (enExample)
MX (1) MX2021005997A (enExample)
PE (1) PE20211419A1 (enExample)
PH (1) PH12021551155A1 (enExample)
SG (1) SG11202105326WA (enExample)
TW (1) TW202039533A (enExample)
WO (1) WO2020106916A1 (enExample)

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CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
US20230285596A1 (en) * 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
US20220106613A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 vector
AU2021353867A1 (en) * 2020-09-29 2023-05-11 NeuExcell Therapeutics Inc. Neurod1 combination vector
US20220098617A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. Ascl1 vector
US20220106614A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Dlx2 vector
US20230392132A1 (en) * 2020-10-21 2023-12-07 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
JP2023553010A (ja) 2020-12-03 2023-12-20 バテル・メモリアル・インスティテュート 非ウイルス性送達のためのポリマーナノ粒子およびdnaナノ構造組成物ならびに方法
AU2022205088A1 (en) * 2021-01-04 2023-07-20 Aavantibio, Inc. Methods and compositions for treatment of friedreich's ataxia
EP4320233A4 (en) 2021-04-07 2025-08-13 Battelle Memorial Institute RAPID DESIGN, BUILD, TEST, AND LEARNING TECHNOLOGIES TO IDENTIFY AND USE NON-VIRAL VECTORS
JP2024534415A (ja) * 2021-09-17 2024-09-20 アステラス ジーン セラピーズ,インコーポレーテッド フラタキシン遺伝子療法
WO2023221942A1 (en) * 2022-05-16 2023-11-23 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating glutaric aciduria type i
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
WO2025122954A1 (en) 2023-12-08 2025-06-12 Battelle Memorial Institute Use of dna origami nanostructures for molecular information based data storage systems
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法

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AU2002254529A1 (en) * 2001-04-04 2002-10-21 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
ES2704677T3 (es) * 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
US10947596B2 (en) * 2014-05-06 2021-03-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators
EP3230441A4 (en) * 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2016115503A1 (en) * 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
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ES2755725T3 (es) * 2015-04-08 2020-04-23 Us Health Terapia génica viral como tratamiento para una enfermedad o un trastorno por almacenamiento de colesterol
WO2016186772A2 (en) * 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
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US10266844B2 (en) * 2015-07-31 2019-04-23 California Institute Of Technology Activity-dependent expression of nucleic acids
RU2021102893A (ru) * 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
CA3008268A1 (en) * 2015-12-15 2017-06-22 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
CA3029119A1 (en) * 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
CA3045335A1 (en) * 2016-12-01 2018-06-07 Universite Laval Crispr-based treatment of friedreich ataxia
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery

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