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DK2002003T3 - Genvektor omfattende miRNA - Google Patents

Genvektor omfattende miRNA Download PDF

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Publication number
DK2002003T3
DK2002003T3 DK06795291.1T DK06795291T DK2002003T3 DK 2002003 T3 DK2002003 T3 DK 2002003T3 DK 06795291 T DK06795291 T DK 06795291T DK 2002003 T3 DK2002003 T3 DK 2002003T3
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vector
gene
expression
viral
cells
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DK06795291.1T
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English (en)
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Luigi Naldini
Brian David Brown
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Ospedale San Raffaele Srl
Fond Telethon
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Publication of DK2002003T3 publication Critical patent/DK2002003T3/da

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Claims (23)

1. Genvektor til anvendelse til behandling, hvilken genvektor omfatter en miRNA-målsekvens og et transgen, som er operativt forbundet til miRNA-målsekvensen, hvor miRNA-målsekvensen er beregnet til forebyggelse eller reduktion af ekspression af transgenet i en celle, som omfatter et tilsvarende endogent miRNA.
2. Genvektor til anvendelse ifølge krav 1, hvilken genvektor foreligger i form af en ikke-viral genvektor.
3. Genvektor til anvendelse ifølge krav 2, hvor den ikke-virale genvektor omfatter en ekspressionsvektor eller et ekspressionspiasmid.
4. Genvektor til anvendelse ifølge krav 1, hvilken genvektor foreligger i form af en viral vektor.
5. Genvektor til anvendelse ifølge krav 4, hvor genvektoren omfatter genomet (RNA eller DNA) fra den virale vektor, hvilket genom omfatter miRNA-målsekvensen.
6. Genvektor til anvendelse ifølge krav 5, hvor genvektoren omfatter genomet fra den virale vektor, hvilket genom omfatter mindst ét transgen, som er operativt forbundet til miRNA-målsekvensen.
7. Genvektor til anvendelse ifølge et hvilket som helst af kravene 4-6, hvor den virale vektor er valgt fra gruppen bestående af retrovirusser, lentivirus, adenovirusser, adenoassocierede virusser, herpes simplex-virus, picornavirusser og alfavirusser.
8. Genvektor til anvendelse ifølge et hvilket som helst af kravene 4-8, hvor den virale vektor kan afledes af et lentivirus.
9. Genvektor til anvendelse ifølge et hvilket som helst af kravene 4-8, hvor den virale vektor foreligger i form af en viral vektorpartikel.
10. Genvektor til anvendelse ifølge et hvilket som helst af kravene 4-9, hvor miRNA-målsekvensen styrer ekspressionen af den virale vektor i en målcelle.
11. Genvektor til anvendelse ifølge et hvilket som helst af de foregående krav, hvilken genvektor omfatter mere end én miRNA-målsekvens, som kan være ens eller forskellige, i tandem-orientering og/eller i forskellige orienteringer.
12. Genvektor til anvendelse ifølge et hvilket som helst af de foregående krav, hvor miRNA-målsekvensen er en, som målsøges af hsa-mir-142as (også kaldet hsa-mir-142-3p)-miRNA, let-7a-, mir-15a-, mir-16-, mir-17-5p-, mir-19-, mir-142-5p-, mir-145- og/eller mir-218-miRNA.
13. Genvektor til anvendelse ifølge et hvilket som helst af de foregående krav, hvor transgenet koder for egnede terapeutiske gener, som indbefatter dem, der koder for tumorsuppressorproteiner, enzymer, prodrugaktiverende enzymer, immunmodulerende molekyler, antistoffer, modificerede immunglobulinlignende molekyler, fusionsproteiner, hormoner, membranproteiner, vasoaktive proteiner eller peptider, cytokiner, kemokiner, anti-virale proteiner, antisense-RNA'er og ribozymer.
14. Genvektor til anvendelse ifølge et hvilket som helst af de foregående krav, hvor vektoren omfatter en vævsspecifik promotor.
15. Farmaceutisk sammensætning til anvendelse til behandling, hvilken sammensætning omfatter genvektoren ifølge et hvilket som helst af kravene 1-14.
16. Celle til anvendelse til behandling, hvilken celle er inficeret eller transduceret med genvektoren ifølge et hvilket som helst af kravene 1-14, hvor cellen ikke er en human embryocelle.
17. Celle til anvendelse ifølge krav 16, hvilken celle er en hæmatopoietisk stamcelle, nyrestamcelle, nervestamcelle, lungestamcelle, leverstamcelle, miltstamcelle, hjertestamcelle, tumorstamcelle eller embryonal stamcelle.
18. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15, hvilken genvektor eller sammensætning omfatter et transgen, som er operativt forbundet til miRNA-målsekvensen, til fremstilling af et lægemiddel til behandling af cancer.
19. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15, hvilken genvektor eller sammensætning omfatter et transgen, som er operativt forbundet til miRNA-målsekvensen, til fremstilling af et lægemiddel til behandling af hæmofili.
20. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15 til fremstilling af et lægemiddel til forebyggelse af immunmedieret afstødning af et overført gen.
21. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15 til fremstilling af et lægemiddel til forebyggelse af immunrespons over for et cirkulerende antigen.
22. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15 til fremstilling af et lægemiddel til behandling af en sygdom valgt blandt cancer, neurologiske sygdomme, arvelige sygdomme, hjertesygdomme, apopleksi, arthritis, virusinfektioner og sygdomme i immunsystemet.
23. Anvendelse af genvektoren ifølge et hvilket som helst af kravene 1-14 eller en farmaceutisk sammensætning ifølge krav 15 til fremstilling af et lægemiddel til genterapi.
DK06795291.1T 2005-05-27 2006-05-26 Genvektor omfattende miRNA DK2002003T3 (da)

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Families Citing this family (140)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006107826A2 (en) 2005-04-04 2006-10-12 The Board Of Regents Of The University Of Texas System Micro-rna's that regulate muscle cells
DK2002003T3 (da) 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Genvektor omfattende miRNA
CA2610702A1 (en) * 2005-06-03 2006-12-07 Michael Zenon Michael Targeting cells with altered microrna expression
AU2007281261B2 (en) 2006-08-01 2013-03-21 Board Of Regents Of The University Of Texas System Identification of a micro-RNA that activates expression of beta-myosin heavy chain
WO2008071959A1 (en) * 2006-12-12 2008-06-19 Oxford Biomedica (Uk) Limited Lentiviral vectors comprising micrornas
EP2125032A4 (en) * 2007-02-20 2011-02-23 Mayo Foundation CANCER TREATMENT WITH VIRAL NUCLEIC ACID
NZ583024A (en) 2007-07-31 2012-04-27 Regents The Univeristy Of Texas System Board Of An agonist of miR-29a, miR-29b or miR-29c that prevents tissue fibrosis and uses thereof
NZ583025A (en) 2007-07-31 2012-06-29 Univ Texas Micro-rnas that control myosin expression and myofiber identity
EP2219653B1 (en) 2007-11-09 2016-12-21 The Board of Regents of The University of Texas System Micro-rnas of the mir-15 family modulate cardiomyocyte survival and cardiac repair
WO2009117418A2 (en) 2008-03-17 2009-09-24 The Board Of Regents Of The University Of Texas System Identification of micro-rnas involved in neuromuscular synapse maintenance and regeneration
CN102099472A (zh) * 2008-05-19 2011-06-15 新加坡科技研究局 使基因表达靶向于神经胶质瘤的核酸分子和方法
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
CN101787373B (zh) * 2009-01-23 2013-06-19 中国人民解放军第二军医大学东方肝胆外科医院 一种携带外源基因在包装细胞中高效生产的重组病毒载体、其构建方法及其用途
WO2010091204A1 (en) 2009-02-04 2010-08-12 Board Of Regents, The University Of Texas System Dual targeting of mir-208 and mir-499 in the treatment of cardiac disorders
US20120115929A1 (en) * 2009-04-29 2012-05-10 Esther Elisa Johanna Maria Creemers Means And Methods For Counteracting, Preventing And/Or Determining Heart Failure, Or A Risk Of Heart Failure
EP2424571B1 (en) 2009-04-30 2020-04-08 Ospedale San Raffaele S.r.l. Gene vector
EP2425001A4 (en) 2009-04-30 2012-11-14 Univ California ANTI-HIV COMBINATION VECTORS, TARGETING VECTORS AND METHOD OF USE
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
DK2459231T3 (da) 2009-07-31 2016-09-05 Ethris Gmbh Rna med en kombination af umodificerede og modificerede nukleotider til proteinekspression
DE102009050308A1 (de) 2009-10-22 2011-05-05 Ludwig-Maximilians-Universität München RNA mit einer Kombination aus unmodifizierten und modifizierten Nucleotiden zur Proteinexpression
WO2011020014A1 (en) 2009-08-14 2011-02-17 Regeneron Pharmaceuticals, Inc. Promoter-regulated differentiation-dependent self-deleting cassette
US9593347B2 (en) * 2010-04-16 2017-03-14 University of Pittsburgh—of the Commonwealth System of Higher Education Identification of mutations in herpes simplex virus envelope glycoproteins that enable or enhance vector retargeting to novel non-HSV receptors
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
EP3536781A1 (en) * 2010-04-23 2019-09-11 University of Massachusetts Cns targeting aav vectors and methods of use thereof
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2012008302A1 (ja) * 2010-07-12 2012-01-19 国立大学法人鳥取大学 ｍｉＲＮＡ導入による新規ｈｉＰＳＣ作製法
WO2012007458A1 (en) 2010-07-12 2012-01-19 Universidad Autónoma De Barcelona Gene therapy composition for use in diabetes treatment
US9248144B2 (en) 2010-11-11 2016-02-02 University Of Miami Compositions, kits and methods for treatment of cardiovascular, immunological and inflammatory diseases
EP2652151A2 (en) 2010-12-15 2013-10-23 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
WO2012143401A1 (en) 2011-04-18 2012-10-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Tissue-specific regulation of transgene expression
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
JP6309450B2 (ja) * 2011-08-04 2018-04-11 イェダ リサーチ アンド デベロップメント カンパニー リミテッド セロトニン関連の医学的状態、アドレナリン関連の医学的状態、ノルアドレナリン関連の医学的状態、グルタミン酸関連の医学的状態および副腎皮質刺激ホルモン放出ホルモン関連の医学的状態の処置および診断のためのミクロｒｎａおよび該ミクロｒｎａを含む組成物
US10130081B2 (en) 2011-08-05 2018-11-20 Regeneron Pharmaceuticals, Inc. Humanized universal light chain mice
WO2013043442A1 (en) * 2011-09-23 2013-03-28 Dharmacon, Inc. The introduction of modular vector elements during production of a lentivirus
KR20140091688A (ko) 2011-10-06 2014-07-22 미라젠 세러퓨틱스 인코포레이티드 마이크로rna 조절에 의한 전신 에너지 항상성의 제어
EP2584040A1 (en) 2011-10-17 2013-04-24 Pharmahungary 2000 Kft. Compounds for treatment of ischemic injury
DE18203666T1 (de) 2012-04-02 2021-10-07 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von sekretierten proteinen
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US20150190532A1 (en) 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
CN103505743A (zh) * 2012-06-21 2014-01-15 北京命码生科科技有限公司 含功能性microRNA/siRNA的细胞微粒子及其应用
EP3354734B1 (en) 2012-06-21 2020-06-10 Miragen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
DK2922554T3 (en) * 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
CA2897941A1 (en) * 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
RS64664B1 (sr) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
AU2014342465B2 (en) 2013-10-28 2018-03-08 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Oncolytic HSV vector
US20150147774A1 (en) * 2013-11-22 2015-05-28 National University Of Singapore Expression construct for yeast and a method of using the construct
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
DK3119797T3 (da) 2014-03-18 2021-03-15 Univ Massachusetts Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
US10975391B2 (en) * 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
WO2016041588A1 (en) 2014-09-16 2016-03-24 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
CA2969599A1 (en) * 2014-12-05 2016-06-09 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
US9885042B2 (en) 2015-01-20 2018-02-06 MiRagen Therapeutics, Inc. miR-92 inhibitors and uses thereof
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
EP4310500A3 (en) 2016-01-15 2024-04-03 American Gene Technologies International Inc. Methods and compositons for the activation of gamma-delta t-cells
PT3408382T (pt) 2016-01-27 2022-06-27 Oncorus Inc Vetores virais oncolíticos e seus usos
MX2018009375A (es) 2016-02-01 2018-09-05 Bioverativ Therapeutics Inc Genes del factor viii optimizados.
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
JP7017247B2 (ja) 2016-03-09 2022-02-08 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド がんを処置するための組み合わせベクターおよび方法
WO2017201342A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. INTEGRATED VIRAL ADMINISTRATION SYSTEM AND RELATED METHODS
JP2019519245A (ja) 2016-06-30 2019-07-11 オンコラス， インコーポレイテッド ポリペプチド製剤の偽型腫瘍溶解性ウィルス送達
IL284348B1 (en) 2016-07-08 2025-02-01 American Gene Tech Int Inc Pre-HIV vaccination and immunotherapy
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
CN110088285A (zh) * 2016-07-26 2019-08-02 森迪生物科学公司 时空调节子
JP2018029521A (ja) * 2016-08-24 2018-03-01 国立研究開発法人農業・食品産業技術総合研究機構 ｍｉＲ３９８標的核酸配列を含む、遺伝子導入のためのウイルスベクター
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
MX2019008105A (es) 2017-01-06 2020-07-22 Stabilitech Biopharma Ltd Virus.
CN107384868A (zh) * 2017-02-17 2017-11-24 中国农业科学院上海兽医研究所 永生化原代绵羊肺细胞系的构建及其应用
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
KR102761577B1 (ko) 2017-05-08 2025-02-03 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 막 융합을 촉진시키기 위한 조성물 및 그의 용도
KR20200003390A (ko) * 2017-05-08 2020-01-09 그릿스톤 온콜로지, 인코포레이티드 알파바이러스 신생항원 벡터
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
EP3621982A4 (en) * 2017-05-12 2021-05-19 University of Massachusetts Viral vector production
PL3652215T3 (pl) 2017-07-14 2021-08-02 Immatics Biotechnologies Gmbh Ulepszona cząsteczka polipeptydu o podwójnej specyficzności
EP3658165A4 (en) 2017-07-26 2021-09-01 Oncorus, Inc. ONCOLYTIC VIRAL VECTORS AND THEIR USES
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
SG11202000764RA (en) 2017-08-09 2020-02-27 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3444347A1 (en) 2017-08-18 2019-02-20 Universität Heidelberg Use of anti-crispr polypeptides for specific activation of cas nucleases
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
EP3694988A4 (en) * 2017-10-10 2021-07-14 NantBio, Inc. MODIFIED EC7 CELLS WITH LOW TOXICITY TO VIRAL PRODUCTION LOADS
CN118345115A (zh) 2017-12-01 2024-07-16 编码治疗公司 工程化的dna结合蛋白
IL275273B2 (en) * 2017-12-15 2024-09-01 Medizinische Hochschule Hannover Improved compound for treatment of heart failure
US11865081B2 (en) 2017-12-29 2024-01-09 Virogin Biotech Canada Ltd. Oncolytic viral delivery of therapeutic polypeptides
SG11202006400UA (en) 2018-01-04 2020-08-28 Iconic Therapeutics Inc Anti-tissue factor antibodies, antibody-drug conjugates, and related methods
CN111918674A (zh) 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
US10716866B2 (en) 2018-03-29 2020-07-21 Generoath Co., Ltd Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
CA3099497A1 (en) 2018-05-15 2019-11-21 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
MX2021000308A (es) 2018-07-09 2021-06-08 Flagship Pioneering Innovations V Inc Composiciones de fusosoma y usos de estas.
JP7602454B2 (ja) 2018-08-09 2024-12-18 バイオベラティブ セラピューティクス インコーポレイテッド 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
BR112021003897A2 (pt) 2018-08-30 2021-05-25 Tenaya Therapeutics, Inc. reprogramação de células cardíacas com miocarina e asci1
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3113325A1 (en) * 2018-10-11 2020-04-16 Eidgenossische Technische Hochschule Zurich A method to treat disease using a nucleic acid vector encoding a highly compact multi-input logic gate
WO2020097049A1 (en) 2018-11-05 2020-05-14 American Gene Technologies International Inc. Vector system for expressing regulatory rna
SG11202105079QA (en) * 2018-11-14 2021-06-29 Flagship Pioneering Innovations V Inc Fusosome compositions for cns delivery
EP3880832A1 (en) 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for hematopoietic stem cell delivery
EP3880180A2 (en) 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
EP3891297A4 (en) * 2018-12-03 2022-09-07 California Institute of Technology In situ readout of dna barcodes
EP3891289A2 (en) 2018-12-06 2021-10-13 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor ix
KR20210107037A (ko) * 2018-12-21 2021-08-31 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 이식유전자 발현의 drg-특이적 감소를 위한 조성물
WO2020142725A1 (en) * 2019-01-04 2020-07-09 Oncorus, Inc. Encapsulated rna polynucleotides and methods of use
KR102248420B1 (ko) * 2019-03-15 2021-05-06 주식회사 제너로스 miR-142-3p의 표적 서열을 포함하는 재조합 벡터
CN114026236A (zh) * 2019-04-24 2022-02-08 宾夕法尼亚州大学信托人 可用于治疗雷特综合征的组合物
CN119876269A (zh) 2019-05-30 2025-04-25 磨石生物公司 经修饰的腺病毒
US20230270885A2 (en) 2019-09-19 2023-08-31 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
KR102461417B1 (ko) * 2019-11-11 2022-11-01 주식회사 온코크로스 아데노바이러스 벡터를 포함하는 전립선암 진단용 조성물
US20230235354A1 (en) * 2020-03-30 2023-07-27 Greffex, Inc. Enhancement of the production of adenoidvirus-based genetransfer vectors
EP4162059A1 (en) * 2020-05-12 2023-04-12 The Trustees of The University of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN115996758A (zh) 2020-06-19 2023-04-21 吉尼松公司 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统
AU2021297965A1 (en) 2020-06-24 2023-02-23 Bioverativ Therapeutics Inc. Methods for the removal of free factor VIII from preparations of lentiviral vectors modified to express said protein
WO2022008711A1 (en) 2020-07-10 2022-01-13 Genethon A novel muscle-specific promoter
KR20230046313A (ko) 2020-08-06 2023-04-05 그릿스톤 바이오, 인코포레이티드 다중에피토프 백신 카세트
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
KR102626543B1 (ko) * 2020-09-14 2024-01-23 주식회사 제너로스 AIMP2-DX2 및 miR-142의 표적 핵산을 포함하는 재조합 벡터
KR20220035693A (ko) * 2020-09-14 2022-03-22 주식회사 제너로스 표적화된 유전자 전달을 위한 아데노-부속 바이러스 벡터
JP7121086B2 (ja) * 2020-09-25 2022-08-17 ウニベルシダッド アウトノマ デ バルセロナ 脂肪組織に形質導入するのに有用なアデノ随伴ウイルスベクター
KR20230110503A (ko) * 2020-09-30 2023-07-24 주식회사 제너로스 AIMP2-DX2 및 선택적으로 miR-142에 대한 표적 서열 및 이의 조성물을 이용한 노인성 황반 질환의 치료방법
US20220372165A1 (en) 2021-05-05 2022-11-24 Immatics Biotechnologies Gmbh Antigen binding proteins specifically binding prame
IL310879A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Expression system in colovirus
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
KR20240067122A (ko) 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
WO2023142040A1 (en) * 2022-01-29 2023-08-03 Virogin Biotech Canada Ltd. Transcriptional and translational dual regulated oncolytic herpes simplex virus vectors
EP4522226A1 (en) * 2022-05-13 2025-03-19 Generoath Co., Ltd. Methods of treating retinal degenerative diseases using aimp2-dx2 and optionally a target sequence for mir-142 and compositions thereof
WO2025024559A1 (en) 2023-07-24 2025-01-30 Strand Therapeutics Inc. Rna-based synthetic circuit for producing engineered immune cells for an extracorporeal cell therapy
US12281311B1 (en) * 2024-02-20 2025-04-22 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS56169586A (en) 1981-05-02 1981-12-26 Tokyo Tanabe Co Ltd Novel beta-galactosidase
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
CA2144040A1 (fr) 1993-07-13 1995-01-26 Michel Perricaudet Vecteurs adenoviraux defectifs et utilisation en therapie genique
WO1995003400A1 (en) 1993-07-23 1995-02-02 Johns Hopkins University School Of Medicine Recombinationally targeted cloning in yeast artificial chromosomes
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
FR2724945B1 (fr) 1994-09-27 1996-12-27 Centre Nat Rech Scient Vecteurs viraux et utilisation en therapie genique
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
FR2730504B1 (fr) 1995-02-13 1997-03-28 Rhone Poulenc Rorer Sa Procede de preparation de genomes d'adenovirus recombinants
US5723315A (en) 1996-08-23 1998-03-03 Genetics Institute, Inc. Secreted proteins and polynucleotides encoding them
DK0968182T3 (da) 1996-08-07 2004-08-16 Darwin Discovery Ltd Hydroxam- og carboxylsyrederivater med MMP- og TNF-inhiberende aktivitet
US6126939A (en) 1996-09-03 2000-10-03 Yeda Research And Development Co. Ltd. Anti-inflammatory dipeptide and pharmaceutical composition thereof
EP1056860B1 (en) * 1998-02-25 2005-02-09 The Dow Chemical Company High affinity humanized anti-tag-72 monoclonal antibodies
CA2321947A1 (en) 1998-02-25 1999-09-02 The Dow Chemical Company High affinity humanized anti-cea monoclonal antibodies
ES2233600T5 (es) * 2000-01-21 2009-06-22 Biovex Limited Cepas de virus del herpes.
WO2003006465A1 (en) 2001-07-13 2003-01-23 Cv Therapeutics, Inc. Partial and full agonist of a adenosine receptors
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
EP2386637B1 (en) * 2001-09-28 2018-05-16 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
US6998391B2 (en) 2002-02-07 2006-02-14 Supergen.Inc. Method for treating diseases associated with abnormal kinase activity
WO2004058749A1 (en) 2002-12-18 2004-07-15 Vertex Pharmaceuticals Incorporated Benzisoxazole derivatives useful as inhibitors of protein kinases
CA2513679A1 (en) 2003-01-17 2004-08-05 University Of Florida Small interference rna gene therapy
WO2004066183A2 (en) 2003-01-22 2004-08-05 European Molecular Biology Laboratory Microrna
WO2004094642A2 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
US8106180B2 (en) 2003-08-07 2012-01-31 Whitehead Institute For Biomedical Research Methods and products for expression of micro RNAs
US20050142581A1 (en) 2003-09-04 2005-06-30 Griffey Richard H. Microrna as ligands and target molecules
WO2005056761A2 (en) * 2003-12-05 2005-06-23 Massachusetts Institute Of Technology REAGENTS AND METHODS FOR IDENTIFICATION OF RNAi PATHWAY GENES AND CHEMICAL MODULATORS OF RNAi
AU2005272816B2 (en) * 2004-08-10 2011-08-11 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides
US20060056344A1 (en) * 2004-09-10 2006-03-16 Interdigital Technology Corporation Seamless channel change in a wireless local area network
US20060265771A1 (en) * 2005-05-17 2006-11-23 Lewis David L Monitoring microrna expression and function
DK2002003T3 (da) * 2005-05-27 2016-03-21 Ospedale San Raffaele Srl Genvektor omfattende miRNA
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
US20090004668A1 (en) * 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation

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