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MX2019007110A - Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos. - Google Patents

Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.

Info

Publication number
MX2019007110A
MX2019007110A MX2019007110A MX2019007110A MX2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A
Authority
MX
Mexico
Prior art keywords
opmd
reagents
treatment
muscular dystrophy
oculopharyngeal muscular
Prior art date
Application number
MX2019007110A
Other languages
English (en)
Inventor
Suhy David
STRINGS-UFOMBAH Vanessa
Original Assignee
Benitec Biopharma Ltd
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 Biopharma Ltd filed Critical Benitec Biopharma Ltd
Publication of MX2019007110A publication Critical patent/MX2019007110A/es

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    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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
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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
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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
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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
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    • 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
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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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    • 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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Abstract

La presente descripción se refiere a reactivos de interferencia de ARN (iARN), tales como microARN de horquilla pequeña (shmiR) y ARN de horquilla pequeña (ARNhp), para el tratamiento de la distrofia muscular oculofaríngea (OPMD), composiciones que los comprenden y uso de estos para tratar individuos que sufren OPMD o que son propensos a sufrirla. La presente descripción también se refiere al uso de los reactivos de iARN combinados con reactivos de reemplazo de PABPN1, tales como construcciones que codifican la proteína PABPN1 funcional, para el tratamiento de la OPMD, composiciones que los comprenden y uso de estos para tratar individuos que sufren OPMD o que son propensos a sufrirla.
MX2019007110A 2016-12-14 2017-12-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos. MX2019007110A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662434312P 2016-12-14 2016-12-14
PCT/AU2017/051385 WO2018107228A1 (en) 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Publications (1)

Publication Number Publication Date
MX2019007110A true MX2019007110A (es) 2019-12-05

Family

ID=62557655

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019007110A MX2019007110A (es) 2016-12-14 2017-12-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.
MX2024003643A MX2024003643A (es) 2016-12-14 2019-06-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y usos de estos.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024003643A MX2024003643A (es) 2016-12-14 2019-06-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y usos de estos.

Country Status (13)

Country Link
US (2) US11142765B2 (es)
EP (1) EP3555294A4 (es)
JP (2) JP7233149B2 (es)
KR (2) KR102495222B1 (es)
CN (2) CN118421624A (es)
AU (2) AU2017377661B2 (es)
BR (1) BR112019012312A8 (es)
CA (1) CA3047154A1 (es)
IL (2) IL304674B2 (es)
MX (2) MX2019007110A (es)
RU (1) RU2763319C2 (es)
WO (1) WO2018107228A1 (es)
ZA (1) ZA202106827B (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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.
SG11202102185RA (en) * 2018-10-17 2021-05-28 Benitec Biopharma Ltd Methods for treating oculopharyngeal muscular dystrophy (opmd)
CA3130221A1 (en) 2019-02-28 2020-09-03 Benitec IP Holdings Inc. Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)
KR20220121844A (ko) * 2019-12-23 2022-09-01 베르사멥 아게 유전자의 발현을 동시에 조절하기 위한 조성물 및 방법
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2453183C (en) * 2001-07-12 2016-05-10 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
MX2009007308A (es) * 2007-01-31 2009-07-15 Basf Plant Science Gmbh Plantas que tienen rasgos relacionados con el rendimiento mejorado y/o resistencia al estres abiotico aumentada y un metodo para obtener las mismas.
AU2008260103B2 (en) * 2007-05-31 2014-04-03 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
WO2010105372A1 (en) * 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
CN104334196B (zh) * 2012-02-16 2018-04-10 Atyr 医药公司 用于治疗自身免疫疾病和炎性疾病的组氨酰‑tRNA合成酶
WO2014077693A1 (en) * 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
AU2014205036A1 (en) * 2013-01-08 2015-07-30 Benitec Biopharma Limited Age-related macular degeneration treatment
SG11201808981XA (en) * 2016-04-14 2018-11-29 Benitec Biopharma Ltd 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.

Also Published As

Publication number Publication date
CN110382698A (zh) 2019-10-25
US11142765B2 (en) 2021-10-12
RU2019121950A3 (es) 2021-01-18
IL267353B2 (en) 2023-12-01
IL304674B2 (en) 2024-11-01
IL267353A (en) 2019-08-29
CA3047154A1 (en) 2018-06-21
US20190330629A1 (en) 2019-10-31
IL267353B1 (en) 2023-08-01
JP2020503014A (ja) 2020-01-30
AU2017377661A1 (en) 2019-07-18
KR102667867B1 (ko) 2024-05-29
BR112019012312A2 (pt) 2020-02-11
BR112019012312A8 (pt) 2023-02-28
WO2018107228A1 (en) 2018-06-21
EP3555294A1 (en) 2019-10-23
AU2023222827A1 (en) 2023-09-14
RU2763319C2 (ru) 2021-12-28
CN110382698B (zh) 2024-05-10
ZA202106827B (en) 2022-07-27
AU2017377661B2 (en) 2023-07-27
KR20230020586A (ko) 2023-02-10
MX2024003643A (es) 2024-04-15
US20220056446A1 (en) 2022-02-24
CN118421624A (zh) 2024-08-02
IL304674B1 (en) 2024-07-01
KR20190104151A (ko) 2019-09-06
IL304674A (en) 2023-09-01
US11932852B2 (en) 2024-03-19
RU2019121950A (ru) 2021-01-18
JP7233149B2 (ja) 2023-03-06
JP7431359B2 (ja) 2024-02-14
AU2023222827B2 (en) 2025-02-27
KR102495222B1 (ko) 2023-02-09
EP3555294A4 (en) 2020-08-26
JP2023062092A (ja) 2023-05-02

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