MX375360B - Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno. - Google Patents

Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno.

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Publication number
MX375360B
MX375360B MX2016006774A MX2016006774A MX375360B MX 375360 B MX375360 B MX 375360B MX 2016006774 A MX2016006774 A MX 2016006774A MX 2016006774 A MX2016006774 A MX 2016006774A MX 375360 B MX375360 B MX 375360B
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MX
Mexico
Prior art keywords
g6pc
storage disease
glycogen storage
coding sequence
intron
Prior art date
Application number
MX2016006774A
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English (en)
Other versions
MX2016006774A (es
Inventor
Barry J Byrne
Janice J Chou
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Us Health
Univ Florida
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 Us Health, Univ Florida filed Critical Us Health
Publication of MX2016006774A publication Critical patent/MX2016006774A/es
Publication of MX375360B publication Critical patent/MX375360B/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
    • C12N15/63—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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/06—Antigout agents, e.g. antihyperuricemic or uricosuric agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
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  • Obesity (AREA)
  • Physics & Mathematics (AREA)
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  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)

Abstract

La presente descripción describe vectores de virus adeno-asociados (AAV) mejorados para aplicaciones de terapia génica en el tratamiento de la enfermedad de almacenamiento de glucógeno, en especial glucogenosis tipo Ia (GSD-Ia). Se describen moléculas recombinantes de ácido nucleico, vectores y AAV recombinante que incluyen un promotor/potenciador G6PC, un intrón sintético, una secuencia codificante G6PC (tal como una secuencia codificante codón-optimizada de G6PC o de tipo salvaje) y la secuencia stuffer o de relleno de ácido nucleico situada entre el promotor/potenciador G6PC y el intrón, así como entre el intrón y la secuencia codificante de G6PC. Los AAV recombinantes descritos en el presente documento muestran transducción altamente eficiente del hígado y son capaces de corregir anormalidades metabólicas en un modelo animal de GSD-Ia.
MX2016006774A 2013-11-26 2014-11-25 Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno. MX375360B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361908861P 2013-11-26 2013-11-26
PCT/US2014/067415 WO2015081101A1 (en) 2013-11-26 2014-11-25 Adeno-associated virus vectors for treatment of glycogen storage disease

Publications (2)

Publication Number Publication Date
MX2016006774A MX2016006774A (es) 2016-10-13
MX375360B true MX375360B (es) 2025-03-06

Family

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Family Applications (1)

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MX2016006774A MX375360B (es) 2013-11-26 2014-11-25 Vectores de virus adeno-asociado para el tratamiento de la enfermedad de almacenamiento de glucógeno.

Country Status (10)

Country Link
US (3) US9644216B2 (es)
EP (2) EP3074510B1 (es)
JP (1) JP6649265B2 (es)
CN (1) CN105934515B (es)
AU (1) AU2014354839B2 (es)
CA (1) CA2930872C (es)
ES (1) ES2690643T3 (es)
IL (1) IL245659B (es)
MX (1) MX375360B (es)
WO (1) WO2015081101A1 (es)

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WO2018140946A1 (en) * 2017-01-30 2018-08-02 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Recombinant virus vectors for the treatment of glycogen storage disease
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CN112334489A (zh) * 2018-05-16 2021-02-05 星火治疗有限公司 密码子优化的酸性α-葡萄糖苷酶表达盒及其使用方法
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CA2930872C (en) 2022-05-31
AU2014354839B2 (en) 2020-06-04
US11060110B2 (en) 2021-07-13
EP3074510B1 (en) 2018-08-01
IL245659A0 (en) 2016-06-30
US20190017069A1 (en) 2019-01-17
AU2014354839A1 (en) 2016-06-09
JP6649265B2 (ja) 2020-02-19
US10113183B2 (en) 2018-10-30
IL245659B (en) 2020-01-30
WO2015081101A1 (en) 2015-06-04
EP3074510A1 (en) 2016-10-05
MX2016006774A (es) 2016-10-13
CA2930872A1 (en) 2015-06-04
ES2690643T3 (es) 2018-11-21
JP2016538885A (ja) 2016-12-15
US9644216B2 (en) 2017-05-09
US20170233763A1 (en) 2017-08-17
CN105934515A (zh) 2016-09-07
EP3415620B1 (en) 2021-03-31
WO2015081101A8 (en) 2016-06-16
BR112016011997A2 (pt) 2017-09-26
EP3415620A1 (en) 2018-12-19
CN105934515B (zh) 2020-08-04
US20160376608A1 (en) 2016-12-29

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