KR102390075B1 - 오르니틴 트랜스카르바밀라아제（ｏｔｃ） 결핍증의 치료에 유용한 조성물 - Google Patents

오르니틴 트랜스카르바밀라아제（ｏｔｃ） 결핍증의 치료에 유용한 조성물 Download PDF

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KR102390075B1
KR102390075B1 KR1020167027891A KR20167027891A KR102390075B1 KR 102390075 B1 KR102390075 B1 KR 102390075B1 KR 1020167027891 A KR1020167027891 A KR 1020167027891A KR 20167027891 A KR20167027891 A KR 20167027891A KR 102390075 B1 KR102390075 B1 KR 102390075B1
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릴리 왕
제임스 엠. 윌슨
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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KR1020167027891A 2014-03-09 2015-03-09 오르니틴 트랜스카르바밀라아제（ｏｔｃ） 결핍증의 치료에 유용한 조성물 Active KR102390075B1 (ko)

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US201461950157P 2014-03-09 2014-03-09
US61/950,157 2014-03-09
PCT/US2015/019513 WO2015138348A1 (en) 2014-03-09 2015-03-09 Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency

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Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9987354B2 (en) 2011-04-29 2018-06-05 Selecta Biosciences, Inc. Tolerogenic synthetic nanocarriers for antigen-specific deletion of T effector cells
CN110251675A (zh) 2013-05-03 2019-09-20 西莱克塔生物科技公司 降低或预防响应于非变应原性抗原的过敏反应的致耐受性合成纳米载体
EP3027221A1 (en) 2013-07-30 2016-06-08 PhaseRx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
WO2015138357A2 (en) * 2014-03-09 2015-09-17 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of otc deficency
WO2016037161A2 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
US11078247B2 (en) 2016-05-04 2021-08-03 Curevac Ag RNA encoding a therapeutic protein
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
AU2017313917B2 (en) 2016-08-18 2023-12-21 The Regents Of The University Of California CRISPR-Cas genome engineering via a modular AAV delivery system
US11400109B2 (en) 2016-11-10 2022-08-02 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
EP3562494A4 (en) * 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
EP3565605A1 (en) * 2017-01-03 2019-11-13 ethris GmbH Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
ES3052719T3 (en) * 2017-01-10 2026-01-13 Childrens Medical Res Institute Polynucleotides and vectors for the expression of transgenes
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
AU2018275549B2 (en) * 2017-05-31 2024-12-19 The University Of North Carolina At Chapel Hill Optimized human clotting factor IX gene expression cassettes and their use
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
JP7423522B2 (ja) * 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
EP3793689A1 (en) * 2018-05-15 2021-03-24 Translate Bio, Inc. Subcutaneous delivery of messenger rna
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
CA3106639A1 (en) * 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
AU2019304569B2 (en) 2018-07-17 2023-07-06 Helixmith Co., Ltd Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms
WO2020033863A1 (en) * 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3122080A1 (en) * 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
KR20210130158A (ko) 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
WO2021178510A1 (en) * 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
JP7817176B2 (ja) 2020-03-04 2026-02-18 ポセイダ セラピューティクス，インコーポレイティド 代謝性肝障害の治療のための組成物と方法
US20230235353A1 (en) * 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
EP4162059A1 (en) 2020-05-12 2023-04-12 The Trustees of The University of Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
JPWO2022224372A1 (enExample) * 2021-04-21 2022-10-27
AR126845A1 (es) * 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
MX2024009027A (es) * 2022-01-21 2024-08-06 Univ Pennsylvania Metodos para el tratamiento de deficiencia de ornitina transcarbamilasa (otc).
JP2025534674A (ja) * 2022-10-11 2025-10-17 パシフィック・ノースウェスト・リサーチ・インスティテュート 遺伝性障害に関する構築物
WO2025229995A1 (ja) * 2024-04-30 2025-11-06 学校法人自治医科大学 核酸導入用ベクター、核酸導入方法、ポリヌクレオチド、及びベクター

Family Cites Families (81)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1016745A (en) 1911-04-11 1912-02-06 Edwin C Henrikson Table-caster.
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
EP0950111A1 (en) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
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
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
EP0938578B1 (en) 1996-12-05 2004-02-04 Crucell Holland B.V. Genetic modification of primate hemopoietic repopulating stem cells
EP2327797B1 (en) 1997-04-01 2015-11-25 Illumina Cambridge Limited Method of nucleic acid sequencing
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
WO1999015677A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Method for gene transfer using bcl2 and compositions useful therein
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
DE69922934T2 (de) 1998-03-20 2005-12-08 The Trustees Of The University Of Pennsylvania Zusammensetzungen und Verfahren zur helfer-freien Herstellung von rekombinanten Adeno-assoziierten Viren
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
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
AU2409200A (en) 1999-06-02 2000-12-28 Trustees Of The University Of Pennsylvania, The Compositions and methods useful for production of recombinant viruses which require helper viruses
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
EP1218035A2 (en) 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Rapid peg-modification of viral vectors
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
JP2003511037A (ja) 1999-10-01 2003-03-25 ジェノボ， インコーポレイテッド ＡＡＶｒｅｐ／ｃａｐ遺伝子を含むアデノウイルスを使用する組換えＡＡＶの産生
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
US6821512B1 (en) 1999-12-03 2004-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals
WO2001040455A2 (en) 1999-12-03 2001-06-07 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
WO2001091802A1 (en) * 2000-05-30 2001-12-06 Baylor College Of Medicine Chimeric viral vectors for gene therapy
AU2001268149B2 (en) 2000-06-01 2005-08-18 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
JP2004520812A (ja) 2000-08-30 2004-07-15 ハプロゲン・エルエルシー 対立遺伝子を決定するための方法
WO2002095073A1 (en) 2001-05-18 2002-11-28 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences
AU2002324700A1 (en) 2001-08-14 2003-03-03 Bayer Ag Nucleic acid and amino acid sequences involved in pain
US20040086485A1 (en) 2001-10-04 2004-05-06 Aguilar-Cordova Carlos Estuardo Chemeric viral vectors for gene therapy
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
PT2573170T (pt) 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
AU2002367943A1 (en) 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2799547B1 (en) 2006-11-08 2016-12-21 Veritas Bio, LLC In Vivo Delivery of RNA to a Target Cell
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
CA2724105C (en) 2008-05-13 2017-09-05 University Of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
EP2352522A4 (en) 2008-11-06 2014-03-05 Univ Washington BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE
EP2364330B1 (en) 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
HRP20140327T4 (hr) 2009-07-24 2017-11-03 Immune Design Corp Lentiviralni vektori pseudotipizirani sa glikoproteinom ovojnice virusa sindbis
EP2281579A1 (en) 2009-08-05 2011-02-09 BioNTech AG Vaccine composition comprising 5'-Cap modified RNA
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG10201908848RA (en) 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9181675B2 (en) 2011-05-12 2015-11-10 Hitachi Construction Machinery Co., Ltd. Construction machine
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
MX363734B (es) 2011-10-27 2019-03-29 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco.
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
HK1206612A1 (en) * 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
EP2834259A4 (en) * 2012-04-02 2016-08-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES
US20150126589A1 (en) 2012-06-08 2015-05-07 Ethris Gmbh Pulmonary Delivery of Messenger RNA
EP3027221A1 (en) * 2013-07-30 2016-06-08 PhaseRx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
WO2015138357A2 (en) 2014-03-09 2015-09-17 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of otc deficency

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Molecular genetics and metabolism, Vol.105, No.2, pp203-211
Molecular Therapy, Vol.17, No. 8, pp1340-1346
Molecular Therapy, Vol.20, No. suppl.1, S56

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