JPWO2022079083A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2022079083A5
JPWO2022079083A5 JP2023523028A JP2023523028A JPWO2022079083A5 JP WO2022079083 A5 JPWO2022079083 A5 JP WO2022079083A5 JP 2023523028 A JP2023523028 A JP 2023523028A JP 2023523028 A JP2023523028 A JP 2023523028A JP WO2022079083 A5 JPWO2022079083 A5 JP WO2022079083A5
Authority
JP
Japan
Prior art keywords
adenovirus
rna
cell
nucleic acid
open reading
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
JP2023523028A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023546116A5 (https=
JP2023546116A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2021/078269 external-priority patent/WO2022079083A1/en
Publication of JP2023546116A publication Critical patent/JP2023546116A/ja
Publication of JP2023546116A5 publication Critical patent/JP2023546116A5/ja
Publication of JPWO2022079083A5 publication Critical patent/JPWO2022079083A5/ja
Pending legal-status Critical Current

Links

JP2023523028A 2020-10-15 2021-10-13 Va rna転写のための核酸構築物 Pending JP2023546116A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20202010 2020-10-15
EP20202010.3 2020-10-15
PCT/EP2021/078269 WO2022079083A1 (en) 2020-10-15 2021-10-13 Nucleic acid constructs for va rna transcription

Publications (3)

Publication Number Publication Date
JP2023546116A JP2023546116A (ja) 2023-11-01
JP2023546116A5 JP2023546116A5 (https=) 2024-10-21
JPWO2022079083A5 true JPWO2022079083A5 (https=) 2024-10-21

Family

ID=72964437

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023523028A Pending JP2023546116A (ja) 2020-10-15 2021-10-13 Va rna転写のための核酸構築物

Country Status (16)

Country Link
US (2) US12312606B2 (https=)
EP (1) EP4229205B1 (https=)
JP (1) JP2023546116A (https=)
KR (1) KR20230085929A (https=)
CN (1) CN116406425A (https=)
AR (1) AR123777A1 (https=)
AU (1) AU2021360197A1 (https=)
CA (1) CA3197730A1 (https=)
ES (1) ES3035361T3 (https=)
HR (1) HRP20250766T1 (https=)
HU (1) HUE071824T2 (https=)
IL (1) IL302046A (https=)
MX (1) MX2023004052A (https=)
PL (1) PL4229205T3 (https=)
TW (1) TW202223094A (https=)
WO (1) WO2022079083A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
WO2025217034A1 (en) * 2024-04-07 2025-10-16 Spark Therapeutics, Inc. Compositions and methods for the regulation of recombinant adeno-associated virus production

Family Cites Families (78)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6093699A (en) 1987-07-09 2000-07-25 The University Of Manitoba Method for gene therapy involving suppression of an immune response
ATE157012T1 (de) 1989-11-03 1997-09-15 Univ Vanderbilt Verfahren zur in vivo-verabreichung von funktionsfähigen fremden genen
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
CA2096222C (en) 1990-11-13 1998-12-29 Stephen D. Lupton Bifunctional selectable fusion genes
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
WO1994017810A1 (en) 1993-02-12 1994-08-18 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
WO1994023744A1 (en) 1993-04-16 1994-10-27 The Wistar Institute Of Anatomy And Biology Recombinant cytomegalovirus vaccine
CN1127527A (zh) 1993-05-17 1996-07-24 加利福尼亚大学董事会 人免疫缺陷型病毒感染和艾滋病的核酶基因治疗方法
WO1994028143A1 (en) 1993-05-21 1994-12-08 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
WO1996026742A1 (en) 1995-02-28 1996-09-06 The Regents Of The University Of California Gene transfer-mediated angiogenesis therapy
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
FR2743818B1 (fr) * 1996-01-23 1998-04-10 Agronomique Inst Nat Rech Constructions d'adn et vecteurs d'expression derives du gene de l'arn va i d'adenovirus
US6228646B1 (en) 1996-03-07 2001-05-08 The Regents Of The University Of California Helper-free, totally defective adenovirus for gene therapy
EP0912873B1 (de) 1996-07-16 2000-12-06 STEYR MANNLICHER Aktiengesellschaft & Co. KG Gewehr mit lösbarer laufbefestigung
JPH1033175A (ja) 1996-07-24 1998-02-10 Hisamitsu Pharmaceut Co Inc 組換えアデノ随伴ウイルスベクターの製造方法
IL128736A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
DE19650714A1 (de) 1996-12-06 1998-06-10 Melchner Harald Von Prof Dr Genfallen-Konstrukt zur Identifizierung und Isolierung von Genen
US6743620B1 (en) 1996-12-16 2004-06-01 Eisai Co., Ltd. Method for preparing retrovirus vector for gene therapy
CA2269661A1 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Recombinase-activatable aav packaging cassettes for use in the production of aav vectors
US6303302B1 (en) 1997-11-19 2001-10-16 The Whitehead Institute For Biomedical Research Regulation of fungal gene expression
AU3097399A (en) 1998-03-20 1999-10-11 Trustees Of The University Of Pennsylvania, The Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6291214B1 (en) 1998-05-11 2001-09-18 Glaxo Wellcome Inc. System for generating recombinant viruses
KR20020057975A (ko) 1999-10-29 2002-07-12 오미야 히사시 유전자 도입 방법
DE19955558C2 (de) 1999-11-18 2003-03-20 Stefan Kochanek Permanente Amniozyten-Zelllinie, ihre Herstellung und Verwendung zur Herstellung von Gentransfervektoren
EP1134287A1 (en) 2000-03-08 2001-09-19 Universite De Geneve A system to control the expression of a given gene using another gene that encodes a polypeptide with recombinant activity
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
CA2416701A1 (en) 2000-07-21 2002-01-31 The United States Of America, As Represented By The Secretary Of Agricul Ture Methods for the replacement, translocation and stacking of dna in eukaryotic genomes
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
AU2002219841A1 (en) 2000-11-16 2002-05-27 Cornell Research Foundation Inc. Vectors for conditional gene inactivation
US7074611B2 (en) 2001-04-27 2006-07-11 Gie-Cerbn, Centre Europeen De Recherche En Biologie Et En Medecine (Gie) Method for the stable inversion of DNA sequence by site-specific recombination and DNA vectors and transgenic cells thereof
DE10210139A1 (de) 2002-03-07 2003-10-23 Medigene Ag Helferkonstrukte für die Herstellung hybrider rAAV-Partikel unterschiedlicher AAV-Serotypen
WO2003084977A1 (en) 2002-04-04 2003-10-16 Xiao Xiao Gene expression control system and its use in recombinant virus packaging cell lines
US7267979B2 (en) 2002-07-01 2007-09-11 Pioneer Hi-Bred International, Inc. Method of controlling gene silencing using site specific recombination
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL BASED RNA INTERFERENCE AND ASSOCIATED METHODS AND COMPOSITIONS
FR2852021B1 (fr) * 2003-03-04 2007-12-07 Methodes et outils pour le criblage d'arn actifs in cellulo
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
US20050130919A1 (en) 2003-07-18 2005-06-16 University Of Massachusetts Regulatable promoters for synthesis of small hairpin RNA
EP1512742A1 (en) 2003-09-08 2005-03-09 Margarete Odenthal Conditional modification of sirnas expression
WO2005116225A1 (en) 2004-05-25 2005-12-08 Children's Hospital Medical Center Method for introducing and expressing rna in cells
DE602004002471T2 (de) 2004-06-22 2007-01-18 Alcatel Verfahren und System zur Beteitstellung einer Übertragungsverbindung für Datenstromverkehr
US20060110390A1 (en) 2004-08-25 2006-05-25 Myogen, Inc. Inhibition of Ku as a treatment for cardiovascular diseases
US7189561B2 (en) 2004-09-21 2007-03-13 Advec, Inc. Production of adenovirus vectors with reduced levels of replication competent adenovirus contamination
DE102005054628A1 (de) 2005-11-16 2007-05-24 Cevec Pharmaceuticals Gmbh Verfahren zur Herstellung von permanenten humanen Zelllinien
AU2006339519A1 (en) 2005-12-16 2007-09-13 The Uab Research Foundation Compositions and methods related to controlled gene expression using viral vectors
US20100173387A1 (en) 2006-04-28 2010-07-08 Universitat Autonoma De Barcelona Method for producing adenovirus vectors for gene therapy and dna sequences used therefor
RS56844B1 (sr) 2007-12-11 2018-04-30 Univ North Carolina Chapel Hill Retrovirusni vektori sa modifikovanim polipurinskim nizom
IL201999A (en) 2009-11-08 2017-10-31 Medical Res & Development Fund For Health Services Bnai Zion Medical Center The State Of Israel A knockout mouse of the 1 – mo gene, a gene associated with morbidly extreme obesity
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
US20130058871A1 (en) 2011-07-28 2013-03-07 Howard Hughes Medical Institute Method and system for mapping synaptic connectivity using light microscopy
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
CN104540382A (zh) 2012-06-12 2015-04-22 弗·哈夫曼-拉罗切有限公司 用于产生条件性敲除等位基因的方法和组合物
DK2931897T3 (en) 2012-12-12 2018-02-05 Broad Inst Inc CONSTRUCTION, MODIFICATION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTICAL APPLICATIONS
US10183978B2 (en) 2013-01-27 2019-01-22 Exemplar Genetics, Llc Animal models of duchenne muscular dystrophy
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
WO2016057800A1 (en) 2014-10-09 2016-04-14 The Regents Of The University Of California Targeted disruption of a csf1-dap12 pathway member gene for the treatment of neuropathic pain
KR102243243B1 (ko) 2014-10-23 2021-04-22 리제너론 파마슈티칼스 인코포레이티드 신규한 cho 통합 부위 및 이의 용도
AU2016362317B2 (en) 2015-12-01 2023-03-16 Spark Therapeutics, Inc. Scalable methods for producing recombinant Adeno-Associated Viral (AAV) vector in serum-free suspension cell culture system suitable for clinical use
WO2017189683A1 (en) 2016-04-26 2017-11-02 Massachusetts Institute Of Technology Extensible recombinase cascades
WO2018096356A1 (en) 2016-11-28 2018-05-31 Horizon Discovery Limited Methods for conditional gene knock-out
WO2018150271A1 (en) * 2017-02-17 2018-08-23 Lonza Ltd. Mammalian cells for producing adeno-associated viruses
AU2018281306B2 (en) 2017-06-07 2025-04-17 Spark Therapeutics, Inc. Enhancing agents for improved cell transfection and/or rAAV vector production
US20200332278A1 (en) 2017-06-16 2020-10-22 Universite De Strasbourg Methods to generate conditional knock-in models
MY207534A (en) 2017-06-30 2025-03-03 Spark Therapeutics Inc Aav vector column purification methods
EP3456822A1 (en) 2017-09-19 2019-03-20 CEVEC Pharmaceuticals GmbH Inducible aav rep genes
IL275462B2 (en) 2017-12-22 2026-01-01 Genentech Inc Targeted integration of nucleic acids
WO2019217483A1 (en) * 2018-05-07 2019-11-14 Spark Therapeutics, Inc. Plasmid free aav vector producing cell lines
GB201816919D0 (en) 2018-10-17 2018-11-28 Glaxosmithkline Ip Dev Ltd Adeno-associated viral vector producer cell lines
SG11202106217TA (en) * 2018-12-21 2021-07-29 Lonza Walkersville Inc Adeno-associated virus (aav) producer cell line and related methods

Similar Documents

Publication Publication Date Title
Kotin Large-scale recombinant adeno-associated virus production
JP6174318B2 (ja) アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物
US6943019B2 (en) Methods and vector constructs useful for production of recombinant AAV
JP7601339B2 (ja) Aavを産生する方法
JP2024009857A5 (https=)
US7851195B2 (en) High-efficiency wild-type-free AAV helper functions
JPWO2020086881A5 (https=)
GB2592751A (en) Process for making adenoassociated viral vectors
US20040209245A1 (en) Production of pseudotyped recombinant AAV virions
IL259595B2 (en) Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
JP7530431B2 (ja) Dna増幅方法
US20230323387A1 (en) Plasmid system
JP2023138527A (ja) スペーサーを含む核酸分子およびその使用の方法
EP4200428B1 (en) Method of making recombinant aavs
US20220242917A1 (en) Compositions and methods for producing adeno-associated viral vectors
CN118460614A (zh) 一种腺相关病毒Cap蛋白的表达框
JPWO2022079083A5 (https=)
JP2025535062A (ja) 二重トランスフェクションベクター
WO2024143440A1 (ja) アデノ随伴ウイルスの製造方法、およびベクターのセット
JPWO2021221995A5 (https=)
JPWO2022223954A5 (https=)
US20250179520A1 (en) Recombinant aav vectors and uses thereof
JPWO2023220502A5 (https=)
KR20260052122A (ko) 아데노-연관 바이러스(AAV) 기반 벡터의 생산을 위한 아데노바이러스 E2a 유전자의 변형된 서열 (변이체)
TW202506203A (zh) 用於產生根據腺相關病毒(AAV)之載體的腺病毒E2a基因的經修飾之序列(變體)