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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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    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

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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EA035829B1 (ru) 2013-08-08 2020-08-18 Глобал Био Терапьютикс, Инк. Зажимное устройство для минимального захвата и его использование
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
CA3205555A1 (en) * 2014-04-25 2015-10-29 Genethon Treatment of hyperbilirubinemia
WO2015191508A1 (en) 2014-06-09 2015-12-17 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
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
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
HK1244299A1 (zh) 2014-11-14 2018-08-03 Voyager Therapeutics, Inc. 治療肌萎縮側索硬化(als)的組合物和方法
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF DISEASES
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
WO2017201346A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3621637A1 (en) 2017-05-09 2020-03-18 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
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US12171843B2 (en) * 2018-02-07 2024-12-24 Genethon Hybrid regulatory elements
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Also Published As

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

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