MX2021004225A - Metodos para tratar distrofia muscular oculofaringea (dmof). - Google Patents

Metodos para tratar distrofia muscular oculofaringea (dmof).

Info

Publication number
MX2021004225A
MX2021004225A MX2021004225A MX2021004225A MX2021004225A MX 2021004225 A MX2021004225 A MX 2021004225A MX 2021004225 A MX2021004225 A MX 2021004225A MX 2021004225 A MX2021004225 A MX 2021004225A MX 2021004225 A MX2021004225 A MX 2021004225A
Authority
MX
Mexico
Prior art keywords
opmd
methods
reagents
muscular dystrophy
oculopharyngeal muscular
Prior art date
Application number
MX2021004225A
Other languages
English (en)
Inventor
David Suhy
Vanessa Strings-Ufombah
Original Assignee
Benitec Ip Holdings Inc
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 Benitec Ip Holdings Inc filed Critical Benitec Ip Holdings Inc
Publication of MX2021004225A publication Critical patent/MX2021004225A/es

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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • 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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    • 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

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  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Organic Chemistry (AREA)
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  • Biotechnology (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente descripción se refiere a métodos para administrar un constructo de terapia génica que comprende reactivos de interferencia de ARN (ARNi), tal como microARN de horquilla corta (shmiR), en combinación con reactivos de reemplazo PABPN1, tal como polinucleótidos que codifican la proteína PABPN1 funcional los cuales no son el objetivo de los reactivos de ARNi, para el tratamiento de distrofia muscular oculofaríngea (DMOF) en individuos que padecen DMOF o que están predispuestos a la misma. En ciertos aspectos, el método comprende la inyección directa a los músculos faríngeos de un sujeto.
MX2021004225A 2018-10-17 2019-10-17 Metodos para tratar distrofia muscular oculofaringea (dmof). MX2021004225A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862747089P 2018-10-17 2018-10-17
PCT/AU2019/051134 WO2020077412A1 (en) 2018-10-17 2019-10-17 Methods for treating oculopharyngeal muscular dystrophy (opmd)

Publications (1)

Publication Number Publication Date
MX2021004225A true MX2021004225A (es) 2021-05-27

Family

ID=70282838

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021004225A MX2021004225A (es) 2018-10-17 2019-10-17 Metodos para tratar distrofia muscular oculofaringea (dmof).

Country Status (12)

Country Link
US (1) US20220106594A1 (es)
EP (1) EP3866859A4 (es)
JP (1) JP2022508835A (es)
KR (1) KR20210081361A (es)
CN (1) CN112867511A (es)
AU (1) AU2019362925A1 (es)
BR (1) BR112021007256A2 (es)
CA (1) CA3114945A1 (es)
IL (1) IL282323A (es)
MX (1) MX2021004225A (es)
SG (1) SG11202102185RA (es)
WO (1) WO2020077412A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014117050A2 (en) * 2013-01-26 2014-07-31 Mirimus, Inc. Modified mirna as a scaffold for shrna
CA3020754C (en) * 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
MX2019007110A (es) * 2016-12-14 2019-12-05 Benitec Biopharma Ltd Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.
WO2020172720A1 (en) * 2019-02-28 2020-09-03 Benitec Biopharma Limited Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)

Also Published As

Publication number Publication date
KR20210081361A (ko) 2021-07-01
EP3866859A1 (en) 2021-08-25
SG11202102185RA (en) 2021-05-28
CA3114945A1 (en) 2020-04-23
CN112867511A (zh) 2021-05-28
JP2022508835A (ja) 2022-01-19
IL282323A (en) 2021-05-31
AU2019362925A1 (en) 2021-05-06
EP3866859A4 (en) 2023-08-09
BR112021007256A2 (pt) 2021-08-10
WO2020077412A1 (en) 2020-04-23
US20220106594A1 (en) 2022-04-07

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