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MX2018015461A - Genes cln1 optimizados y cassettes de expresion y su uso. - Google Patents

Genes cln1 optimizados y cassettes de expresion y su uso.

Info

Publication number
MX2018015461A
MX2018015461A MX2018015461A MX2018015461A MX2018015461A MX 2018015461 A MX2018015461 A MX 2018015461A MX 2018015461 A MX2018015461 A MX 2018015461A MX 2018015461 A MX2018015461 A MX 2018015461A MX 2018015461 A MX2018015461 A MX 2018015461A
Authority
MX
Mexico
Prior art keywords
genes
expression cassettes
optimized
cln1
reading frame
Prior art date
Application number
MX2018015461A
Other languages
English (en)
Inventor
Gray Steven
Original Assignee
Univ North Carolina Chapel Hill
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 Univ North Carolina Chapel Hill filed Critical Univ North Carolina Chapel Hill
Publication of MX2018015461A publication Critical patent/MX2018015461A/es

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    • 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
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    • C12Y301/02—Thioester hydrolases (3.1.2)
    • C12Y301/02022—Palmitoyl-protein hydrolase (3.1.2.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
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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)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor

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

Abstract

Esta invención está relacionada con polinucleótidos que comprenden una secuencia de nucleótidos que codifica un polipéptido de PPT1 o un fragmento del mismo, vectores (vectores virales y no virales) que comprenden la misma, y métodos para utilizar la misma para el suministro del marco de lectura abierto a una célula o un sujeto y para tratar lipofuscinosis neuronal infantil (enfermedad de Batten infantil); los polinucleótidos comprenden un marco de lectura abierto de CLN1 optimizado.
MX2018015461A 2016-06-13 2017-06-13 Genes cln1 optimizados y cassettes de expresion y su uso. MX2018015461A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662349411P 2016-06-13 2016-06-13
PCT/US2017/037118 WO2017218450A1 (en) 2016-06-13 2017-06-13 Optimized cln1 genes and expression cassettes and their use

Publications (1)

Publication Number Publication Date
MX2018015461A true MX2018015461A (es) 2019-05-27

Family

ID=60664168

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018015461A MX2018015461A (es) 2016-06-13 2017-06-13 Genes cln1 optimizados y cassettes de expresion y su uso.

Country Status (11)

Country Link
US (2) US11504435B2 (es)
EP (1) EP3469080A4 (es)
JP (1) JP2019517274A (es)
KR (1) KR102629418B1 (es)
CN (1) CN109415730A (es)
AU (1) AU2017284198B2 (es)
CA (1) CA3024536A1 (es)
IL (1) IL263009B2 (es)
MX (1) MX2018015461A (es)
RU (1) RU2764919C2 (es)
WO (1) WO2017218450A1 (es)

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FI3814513T3 (fi) * 2018-06-28 2025-05-28 Univ North Carolina Chapel Hill Optimoidut cln5-geenit ja -ilmentymiskasetit sekä niiden käyttö
EA202192328A1 (ru) * 2019-03-22 2022-02-21 Раш Юниверсити Медикал Сентер Комбинация назальной доставки генов и пероральной доставки коричной кислоты, олеамида или гемфиброзила для лечения лизосомных болезней накопления
JP7788594B2 (ja) * 2019-04-29 2025-12-19 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 乳児バッテン病の治療のための髄腔内および静脈内組合せ遺伝子療法
EP3969059A1 (en) * 2019-05-17 2022-03-23 Research Institute at Nationwide Children's Hospital Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme
US12188039B2 (en) * 2019-11-08 2025-01-07 The Board Of Regents Of The University Of Texas System Recombinant adeno-associated viral vector for gene delivery
WO2021102250A1 (en) * 2019-11-21 2021-05-27 Luppino Francesco Growth factor restoration
WO2021133871A2 (en) * 2019-12-24 2021-07-01 Asklepios Biopharmaceutical, Inc. Method for identifying regulatory elements
EP4185700B1 (en) * 2020-07-23 2025-12-17 The University of North Carolina at Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
CN119971081B (zh) * 2025-02-11 2025-11-18 中央民族大学 用于治疗智力障碍的组合物、其应用以及药物筛选方法

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Also Published As

Publication number Publication date
KR102629418B1 (ko) 2024-01-25
KR20190016509A (ko) 2019-02-18
US11504435B2 (en) 2022-11-22
CN109415730A (zh) 2019-03-01
IL263009A (en) 2018-12-31
BR112018075692A2 (pt) 2019-04-02
RU2018145010A (ru) 2020-07-14
US20200316222A1 (en) 2020-10-08
JP2019517274A (ja) 2019-06-24
AU2017284198B2 (en) 2023-09-28
US12257322B2 (en) 2025-03-25
RU2764919C2 (ru) 2022-01-24
RU2018145010A3 (es) 2020-12-23
EP3469080A4 (en) 2020-01-22
WO2017218450A1 (en) 2017-12-21
IL263009B2 (en) 2023-06-01
US20230372544A1 (en) 2023-11-23
EP3469080A1 (en) 2019-04-17
CA3024536A1 (en) 2017-12-21
AU2017284198A1 (en) 2018-12-06

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