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

Genes cln1 optimizados y cassettes de expresion y su uso.

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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
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MX
Mexico
Prior art keywords
genes
expression cassettes
optimized
cln1
reading frame
Prior art date
Application number
MX2018015461A
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English (en)
Inventor
Gray Steven
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Univ North Carolina Chapel Hill
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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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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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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 (12)

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US (2) US11504435B2 (es)
EP (1) EP3469080A4 (es)
JP (1) JP2019517274A (es)
KR (1) KR102629418B1 (es)
CN (1) CN109415730A (es)
AU (1) AU2017284198B2 (es)
BR (1) BR112018075692A2 (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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US20210269829A1 (en) * 2018-06-28 2021-09-02 The University Of North Carolina At Chapel Hill Optimized cln5 genes and expression cassettes and their use
EP3941584A4 (en) * 2019-03-22 2022-11-30 Rush University Medical Center COMBINATION OF NASAL GENE DELIVERY AND ORAL CINNAID, OLEAMIDE OR GEMFIBROCIL FOR LYSOSOMAL STORAGE DISEASES
JP2022530264A (ja) * 2019-04-29 2022-06-28 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
US20210139933A1 (en) * 2019-11-08 2021-05-13 The Board Of Regents Of The University Of Texas System Recombinant adeno-associated viral vector for gene delivery
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WO2021133871A2 (en) * 2019-12-24 2021-07-01 Asklepios Biopharmaceutical, Inc. Method for identifying regulatory elements
JP2023536067A (ja) * 2020-07-23 2023-08-23 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 最適化されたｓｌｃ１３ａ５遺伝子および発現カセットならびにそれらの使用

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Also Published As

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

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