MX2011003229A - Terapia genica con porfobilinogeno desaminasa. - Google Patents

Terapia genica con porfobilinogeno desaminasa.

Info

Publication number
MX2011003229A
MX2011003229A MX2011003229A MX2011003229A MX2011003229A MX 2011003229 A MX2011003229 A MX 2011003229A MX 2011003229 A MX2011003229 A MX 2011003229A MX 2011003229 A MX2011003229 A MX 2011003229A MX 2011003229 A MX2011003229 A MX 2011003229A
Authority
MX
Mexico
Prior art keywords
porphobilinogen deaminase
coding
codons
gene therapy
human
Prior art date
Application number
MX2011003229A
Other languages
English (en)
Inventor
Antonio Fontanellas Roma
Gloria Gonzalez Aseguinolaza
Maria Sol Rodriguez Pena
Maria Astrid Paneda Rodriguez
Jesus Maria Prieto Valtuena
Jaap Twisk
Harald Petry
Sander Jan Hendrik Van Deventer
Original Assignee
Proyecto Biomedicina Cima Sl
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 Proyecto Biomedicina Cima Sl filed Critical Proyecto Biomedicina Cima Sl
Publication of MX2011003229A publication Critical patent/MX2011003229A/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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88—Lyases (4.)
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/01061—Hydroxymethylbilane synthase (2.5.1.61)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Abstract

La presente invención se refiere a secuencias de nucleótidos que codifican la porfobilinogeno desaminasa que se optimizan para la expresión superior en células mamíferas. La invención se refiere además a constructos de ADN que comprenden tales secuencias optimizadas de codificación sintética para uso en terapia génica de afecciones provocadas por una deficiencia en la porfobilinogeno desaminasa, tal como porfiria intermitente aguda. Consecuentemente, la presente invención se refiere a un ácido nucleico o un constructo de ácido nucleico que comprende una secuencia de nucleótidos que codifica una porfobilinogeno desaminasa humana, en donde al menos 320 de los codones que codifican la porfobilinogeno desaminasa humana son idénticos a los codones en la SEQ ID NO. 1, o en donde al menos 305 de los codones que codifican la porfobilinogeno desaminasa humana son idénticos a los codones en la SEQ ID NO. 3.
MX2011003229A 2008-09-29 2009-09-29 Terapia genica con porfobilinogeno desaminasa. MX2011003229A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10088108P 2008-09-29 2008-09-29
EP08165393 2008-09-29
PCT/NL2009/050584 WO2010036118A1 (en) 2008-09-29 2009-09-29 Porphobilinogen deaminase gene therapy

Publications (1)

Publication Number Publication Date
MX2011003229A true MX2011003229A (es) 2011-09-06

Family

ID=40130866

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2011003229A MX2011003229A (es) 2008-09-29 2009-09-29 Terapia genica con porfobilinogeno desaminasa.

Country Status (12)

Country Link
US (1) US8697665B2 (es)
EP (1) EP2352823B1 (es)
JP (1) JP2012503980A (es)
KR (1) KR20110086553A (es)
CN (1) CN102227500B (es)
AU (1) AU2009297243B2 (es)
CA (1) CA2738969A1 (es)
EA (1) EA201170506A1 (es)
ES (1) ES2507540T3 (es)
MX (1) MX2011003229A (es)
WO (1) WO2010036118A1 (es)
ZA (1) ZA201102287B (es)

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EA033803B1 (ru) 2012-02-07 2019-11-27 Global Bio Therapeutics Inc Способ доставки нуклеиновых кислот с разделением, их композиции и использование
MX2016001667A (es) 2013-08-08 2016-11-07 Global Bio Therapeutics Inc Dispositivo de sujecion para procedimientos minimamente invasivos y usos del mismo.
CN103667346B (zh) * 2013-12-17 2016-01-20 扬州大学 一种pRBE-HCR-hAAT-hFIXml质粒及其构建和应用
CN106459932B (zh) * 2014-04-25 2022-01-11 吉尼松公司 高胆红素血症的治疗
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016037931A1 (en) 2014-09-11 2016-03-17 Fundación Para La Investigación Médica Aplicada Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017201346A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
MA45032A (fr) * 2016-05-18 2019-03-27 Fundacion Para La Investig Medica Aplicada Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518258A (ja) 2017-05-05 2020-06-25 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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
US11390854B2 (en) 2017-05-09 2022-07-19 Fundacion Para La Investigacion Medica Aplicada Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
GB201916983D0 (en) * 2019-11-21 2020-01-08 Vestlandets Innovasjonsselskap As Therapy

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US5974159A (en) * 1996-03-29 1999-10-26 Sarnoff Corporation Method and apparatus for assessing the visibility of differences between two image sequences
CA2319096C (en) 1998-01-27 2007-07-03 Hemebiotech A/S Method for treating acute intermittent porphyria (aip) and other porphyric diseases
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ES2721804T3 (es) * 2006-03-13 2019-08-05 Agc Glass Europe Hoja de vidrio revestida
EP2004693B1 (en) * 2006-03-30 2012-06-06 Novartis AG Compositions and methods of use for antibodies of c-met

Also Published As

Publication number Publication date
AU2009297243A1 (en) 2010-04-01
JP2012503980A (ja) 2012-02-16
WO2010036118A1 (en) 2010-04-01
ES2507540T3 (es) 2014-10-15
EP2352823B1 (en) 2014-08-06
CN102227500B (zh) 2013-05-29
US8697665B2 (en) 2014-04-15
AU2009297243B2 (en) 2015-10-29
KR20110086553A (ko) 2011-07-28
US20110262399A1 (en) 2011-10-27
ZA201102287B (en) 2011-12-28
EP2352823A1 (en) 2011-08-10
CN102227500A (zh) 2011-10-26
EA201170506A1 (ru) 2011-12-30
CA2738969A1 (en) 2010-04-01

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