JP2022508182A5 - - Google Patents

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Publication number
JP2022508182A5
JP2022508182A5 JP2021529002A JP2021529002A JP2022508182A5 JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5
Authority
JP
Japan
Prior art keywords
sequence
nucleic acid
aav vector
acid according
recombinant aav
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.)
Granted
Application number
JP2021529002A
Other languages
English (en)
Japanese (ja)
Other versions
JP7704998B2 (ja
JP2022508182A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/062531 external-priority patent/WO2020106916A1/en
Publication of JP2022508182A publication Critical patent/JP2022508182A/ja
Publication of JP2022508182A5 publication Critical patent/JP2022508182A5/ja
Priority to JP2025034621A priority Critical patent/JP2025102783A/ja
Application granted granted Critical
Publication of JP7704998B2 publication Critical patent/JP7704998B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
JP2022508182A5 true JP2022508182A5 (enExample) 2022-11-30
JP7704998B2 JP7704998B2 (ja) 2025-07-09

Family

ID=70773595

Family Applications (2)

Application Number Title Priority Date Filing Date
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)

Application Number Title Priority Date Filing Date
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
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
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
JP2023542614A (ja) * 2020-08-19 2023-10-11 サレプタ セラピューティクス, インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
EP4221761A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. Neurod1 combination vector
WO2022072309A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Ascl1 vector
MX2023003655A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector neurod1.
CA3197320A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Dlx2 vector
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
IL303390A (en) 2020-12-03 2023-08-01 Battelle Memorial Institute Compositions of polymer nanoparticles and DNA nanostructures and methods for non-viral transport
US20240058477A1 (en) * 2021-01-04 2024-02-22 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
WO2022216977A1 (en) 2021-04-07 2022-10-13 Batelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
MX2024003241A (es) * 2021-09-17 2024-05-15 Astellas Gene Therapies Inc Terapia genica con frataxina.
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
EP4536286A1 (en) * 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE 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
AU2024393766A1 (en) 2023-12-08 2026-06-18 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
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

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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
DK2986635T3 (en) * 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
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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
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EP3280451B8 (en) * 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
PL3298134T3 (pl) * 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
EP4378487A3 (en) * 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2017023708A1 (en) * 2015-07-31 2017-02-09 California Institute Of Technology Activity-dependent expression of nucleic acids
SG10201912763QA (en) * 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
SG10201912935WA (en) * 2015-12-15 2020-02-27 Genzyme Corp 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
US20200056206A1 (en) * 2016-12-01 2020-02-20 UNIVERSITé 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
MX2020003042A (es) * 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

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