MX2019011040A - Composiciones y metodos para potenciar la expresion genica. - Google Patents

Composiciones y metodos para potenciar la expresion genica.

Info

Publication number
MX2019011040A
MX2019011040A MX2019011040A MX2019011040A MX2019011040A MX 2019011040 A MX2019011040 A MX 2019011040A MX 2019011040 A MX2019011040 A MX 2019011040A MX 2019011040 A MX2019011040 A MX 2019011040A MX 2019011040 A MX2019011040 A MX 2019011040A
Authority
MX
Mexico
Prior art keywords
methods
compositions
gene expression
enhanced gene
expression
Prior art date
Application number
MX2019011040A
Other languages
English (en)
Inventor
Keravala Annahita
Original Assignee
Adverum Biotechnologies Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of MX2019011040A publication Critical patent/MX2019011040A/es

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

La presente descripcion proporciona casetes de polinucleotidos, vectores de expresion y metodos para la expresion de un gen en celulas de mamifero.
MX2019011040A 2017-03-17 2018-03-16 Composiciones y metodos para potenciar la expresion genica. MX2019011040A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762472892P 2017-03-17 2017-03-17
PCT/US2018/022996 WO2018170473A1 (en) 2017-03-17 2018-03-16 Compositions and methods for enhanced gene expression

Publications (1)

Publication Number Publication Date
MX2019011040A true MX2019011040A (es) 2020-01-20

Family

ID=63523675

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019011040A MX2019011040A (es) 2017-03-17 2018-03-16 Composiciones y metodos para potenciar la expresion genica.

Country Status (13)

Country Link
US (3) US11773406B2 (es)
EP (1) EP3596213A4 (es)
JP (2) JP7343903B2 (es)
KR (1) KR102616820B1 (es)
CN (1) CN110546257B (es)
AU (1) AU2018234918B2 (es)
BR (1) BR112019019158A2 (es)
CA (1) CA3054942A1 (es)
EA (1) EA201992001A1 (es)
IL (1) IL268788A (es)
MX (1) MX2019011040A (es)
SG (1) SG11201907653QA (es)
WO (1) WO2018170473A1 (es)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
CN107405507B (zh) 2015-03-02 2022-05-03 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
EP3830261A1 (en) * 2018-08-02 2021-06-09 Novozymes A/S Preparation of combinatorial libraries of dna constructs using introns
EP3934698A1 (en) 2019-03-04 2022-01-12 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
US20210100856A1 (en) * 2019-09-11 2021-04-08 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
WO2021050649A1 (en) 2019-09-11 2021-03-18 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
AU2019465527A1 (en) * 2019-09-11 2022-03-10 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using AAV2 variants encoding aflibercept
KR102346159B1 (ko) * 2020-02-20 2022-01-03 국립암센터 고효율 발현 벡터 및 이의 용도
US20220010332A1 (en) * 2020-07-08 2022-01-13 Neuracle Genetics Inc. Intron fragments
CN113817775B (zh) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 修饰的阿柏西普、组合物、方法及其在基因治疗中的应用
US20240117377A1 (en) * 2021-02-02 2024-04-11 Allen Institute Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
CA3215855A1 (en) 2021-04-27 2022-11-03 Julie Clark Methods of treating ocular diseases using aav2 variants encoding aflibercept
WO2023283649A1 (en) * 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
WO2023147604A2 (en) * 2022-01-31 2023-08-03 Trames Bio, Inc. Expression cassettes for treating epilepsy and neuropathic pain
WO2023150566A1 (en) 2022-02-02 2023-08-10 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
WO2023158990A1 (en) 2022-02-16 2023-08-24 Adverum Biotechnologies, Inc. Method of reducing cst fluctuation in neovascular amd by a recombinant adeno-associated virus
WO2023225455A2 (en) * 2022-05-18 2023-11-23 Visgenx, Inc. Synthetic aav genomes for improved gene delivery

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
CA2158745C (en) 1993-03-25 2007-06-19 Richard L. Kendall Inhibitor of vascular endothelial cell growth factor
US20020168342A1 (en) 1994-11-03 2002-11-14 Cell Genesys, Inc. Novel adenoviral vectors, packaging cell lines, recombinant adenoviruses and methods
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6087129A (en) 1996-01-19 2000-07-11 Betagene, Inc. Recombinant expression of proteins from secretory cell lines
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
US8021875B2 (en) 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
WO2004007664A2 (en) * 2002-05-28 2004-01-22 Maxygen, Inc. Nucleic acid vectors
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
AR043017A1 (es) 2003-02-03 2005-07-13 Japan Immuno Inc Vector de alta expresion para celulas animales
US7186699B2 (en) 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1644508A1 (en) 2003-07-11 2006-04-12 Cytos Biotechnology AG Gene expression system
AU2006338570B2 (en) 2005-11-18 2013-07-11 The Ohio State University Research Foundation Viral gene products and methods for vaccination to prevent viral associated diseases
KR101541935B1 (ko) 2007-09-26 2015-08-05 인트렉손 코포레이션 합성 5'ｕｔｒ, 발현 벡터, 및 전이유전자 발현의 증가방법
EP2784089A1 (en) 2008-01-15 2014-10-01 AbbVie Inc. Improved mammalian expression vectors and uses thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA2833870C (en) 2011-04-22 2020-03-10 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
TWI702955B (zh) 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ）
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
JP6719381B2 (ja) 2014-02-06 2020-07-08 ジェンザイム・コーポレーション 黄斑変性を処置し、予防するための組成物および方法
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US10927384B2 (en) 2014-04-09 2021-02-23 Dna Twopointo Inc. DNA vectors, transposons and transposases for eukaryotic genome modification
EP3670660A1 (en) 2014-04-09 2020-06-24 Dna Twopointo Inc. Enhanced nucleic acid constructs for eukaryotic gene expression
JP6401871B2 (ja) * 2014-11-05 2018-10-10 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
CN107405507B (zh) 2015-03-02 2022-05-03 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
JP6544565B2 (ja) 2015-04-28 2019-07-17 国立大学法人広島大学 哺乳動物細胞内で目的遺伝子の発現を高める方法およびキット、並びに、その利用
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
ES2908467T3 (es) 2016-04-29 2022-04-29 Adverum Biotechnologies Inc Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante
WO2017218974A2 (en) 2016-06-16 2017-12-21 Adverum Biotechnologies, Inc. Treatment of amd using aav2 variant with aflibercept
PL3472317T3 (pl) 2016-06-16 2022-08-08 Adverum Biotechnologies, Inc. Kompozycje i sposoby zmniejszania neowaskularyzacji oka
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US20210130413A1 (en) 2017-02-28 2021-05-06 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법

Also Published As

Publication number Publication date
EA201992001A1 (ru) 2020-01-24
WO2018170473A1 (en) 2018-09-20
EP3596213A4 (en) 2021-02-17
IL268788A (en) 2019-10-31
AU2018234918A1 (en) 2019-09-12
EP3596213A1 (en) 2020-01-22
US20210040501A1 (en) 2021-02-11
CA3054942A1 (en) 2018-09-20
US11773406B2 (en) 2023-10-03
SG11201907653QA (en) 2019-09-27
US20200010851A1 (en) 2020-01-09
US20230257777A1 (en) 2023-08-17
KR20190132626A (ko) 2019-11-28
KR102616820B1 (ko) 2023-12-21
JP7343903B2 (ja) 2023-09-13
NZ756504A (en) 2023-11-24
US11352644B2 (en) 2022-06-07
CN110546257B (zh) 2024-03-01
JP2020513792A (ja) 2020-05-21
AU2018234918B2 (en) 2023-11-02
JP2022092057A (ja) 2022-06-21
CN110546257A (zh) 2019-12-06
JP7360208B2 (ja) 2023-10-12
BR112019019158A2 (pt) 2020-05-05

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