CN110382698B - 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 - Google Patents

用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 Download PDF

Info

Publication number
CN110382698B
CN110382698B CN201780086338.5A CN201780086338A CN110382698B CN 110382698 B CN110382698 B CN 110382698B CN 201780086338 A CN201780086338 A CN 201780086338A CN 110382698 B CN110382698 B CN 110382698B
Authority
CN
China
Prior art keywords
shmir
seq
sequence
nucleic acid
effector
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CN201780086338.5A
Other languages
English (en)
Chinese (zh)
Other versions
CN110382698A (zh
Inventor
V·斯特林斯-尤弗姆巴
D·苏伊
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Benitec IP Holdings Inc
Original Assignee
Benitec Biopharma Ltd
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 Biopharma Ltd, Benitec IP Holdings Inc filed Critical Benitec Biopharma Ltd
Priority to CN202410482087.4A priority Critical patent/CN118421624A/zh
Publication of CN110382698A publication Critical patent/CN110382698A/zh
Application granted granted Critical
Publication of CN110382698B publication Critical patent/CN110382698B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N15/11—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
    • 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
    • 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
    • C12N15/861—Adenoviral vectors
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N15/867—Retroviral vectors
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
CN201780086338.5A 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途 Active CN110382698B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410482087.4A CN118421624A (zh) 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662434312P 2016-12-14 2016-12-14
US62/434,312 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202410482087.4A Division CN118421624A (zh) 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途

Publications (2)

Publication Number Publication Date
CN110382698A CN110382698A (zh) 2019-10-25
CN110382698B true CN110382698B (zh) 2024-05-10

Family

ID=62557655

Family Applications (2)

Application Number Title Priority Date Filing Date
CN201780086338.5A Active CN110382698B (zh) 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途
CN202410482087.4A Pending CN118421624A (zh) 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN202410482087.4A Pending CN118421624A (zh) 2016-12-14 2017-12-14 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途

Country Status (12)

Country Link
US (2) US11142765B2 (https=)
EP (1) EP3555294A4 (https=)
JP (2) JP7233149B2 (https=)
KR (2) KR102667867B1 (https=)
CN (2) CN110382698B (https=)
AU (2) AU2017377661B2 (https=)
CA (1) CA3047154A1 (https=)
IL (2) IL267353B2 (https=)
MX (2) MX2019007110A (https=)
RU (1) RU2763319C2 (https=)
WO (1) WO2018107228A1 (https=)
ZA (1) ZA202106827B (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3555294A4 (en) * 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE
CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
US20220098614A1 (en) 2019-02-28 2022-03-31 Benitec IP Holdings Inc. Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD)
CN114901822A (zh) * 2019-12-23 2022-08-12 维萨梅布有限公司 用于同时调节基因表达的组合物和方法
WO2026011009A1 (en) * 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN105143450A (zh) * 2013-01-08 2015-12-09 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2345720A3 (en) * 2001-07-12 2012-01-25 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
AU2008209677B2 (en) * 2007-01-31 2013-05-23 Basf Plant Science Gmbh Plants having enhanced yield-related traits and/or increased abiotic stress resistance, and a method for making the same
US8258286B2 (en) * 2007-04-26 2012-09-04 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
MX356107B (es) * 2012-02-16 2018-05-15 Atyr Pharma Inc Histidil-arnt sintetasas para tratar enfermedades autoinmunes e inflamatorias.
CA3020754C (en) * 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
EP3555294A4 (en) 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN105143450A (zh) * 2013-01-08 2015-12-09 贝尼泰克生物制药有限公司 年龄相关性黄斑变性治疗

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
A decline in PABPN1 induces progressive muscle weakness in Oculopharyngeal muscle dystrophy and in muscle aging;Seyed Yahya Anvar等;《Aging》;20130630;第5卷(第6期);第413页左栏第1段-第3段，第418页右栏第1段，第423页右栏第4段 *
Adeno-associated virus（AAV）-mediated RNA interference to PABPN1 combined with an potimised resistant transgene for rescue of the muscle specific disease oculopharyngeal muscular dytrophy(OPMD);Houria bachtarzi等;《Abstract of the UK Neuromuscular Translation Research Conference 2014》;20140228;全文 *
Loss of nuclear poly(A)-binding protein 1 causes defects in myogenesis and mRNA biogenesis;Apponi, LH等;《HUMAN MOLECULAR GENETICS》;20100315;第19卷(第6期);全文 *
Progress on gene therapy, cell therapy and pharmacological strategies towards the treatment of Oculopharyngeal Muscular Dystrophy;Pradeep Harish等;《Hum Gene Ther》;20150511;第26卷(第5期);第8页第3段 *

Also Published As

Publication number Publication date
JP7431359B2 (ja) 2024-02-14
AU2023222827A1 (en) 2023-09-14
KR20230020586A (ko) 2023-02-10
IL267353B2 (en) 2023-12-01
WO2018107228A1 (en) 2018-06-21
JP2023062092A (ja) 2023-05-02
IL304674B1 (en) 2024-07-01
BR112019012312A8 (pt) 2023-02-28
CN118421624A (zh) 2024-08-02
JP2020503014A (ja) 2020-01-30
US20190330629A1 (en) 2019-10-31
RU2763319C2 (ru) 2021-12-28
RU2019121950A3 (https=) 2021-01-18
MX2024003643A (es) 2024-04-15
AU2017377661A1 (en) 2019-07-18
IL304674A (en) 2023-09-01
KR20190104151A (ko) 2019-09-06
JP7233149B2 (ja) 2023-03-06
CA3047154A1 (en) 2018-06-21
IL304674B2 (en) 2024-11-01
ZA202106827B (en) 2022-07-27
KR102495222B1 (ko) 2023-02-09
BR112019012312A2 (pt) 2020-02-11
KR102667867B1 (ko) 2024-05-29
EP3555294A4 (en) 2020-08-26
IL267353A (en) 2019-08-29
US20220056446A1 (en) 2022-02-24
CN110382698A (zh) 2019-10-25
US11142765B2 (en) 2021-10-12
MX2019007110A (es) 2019-12-05
AU2023222827B2 (en) 2025-02-27
RU2019121950A (ru) 2021-01-18
AU2017377661B2 (en) 2023-07-27
EP3555294A1 (en) 2019-10-23
US11932852B2 (en) 2024-03-19
IL267353B1 (en) 2023-08-01

Similar Documents

Publication Publication Date Title
JP7431359B2 (ja) 眼咽頭型筋ジストロフィー（ｏｐｍｄ）を治療するための試薬及びその使用
KR102353847B1 (ko) 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도
US20220106594A1 (en) Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD)
BR122025028670A2 (pt) Ácido nucleico que codifica micro-rna hairpin curto, construto de interferência de rna, construto de dna, vetor de expressão, combinação de vetores de expressão, composição, método in vitro de inibição da expressão de uma proteína pabpn1, kit e uso
BR112019012312B1 (pt) Ácido nucleico que codifica micro-rna hairpin curto, construto de interferência de rna, construto de dna, vetorde expressão, combinação de vetores, composição, método in vitro de inibição da expressão de uma proteína pabpn1, kit eusos

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
CB02 Change of applicant information
CB02 Change of applicant information

Address after: New South Wales Australia

Applicant after: Benetec Bio-Pharmaceuticals

Address before: New South Wales Australia

Applicant before: BENITEC BIOPHARMA LTD.

TA01 Transfer of patent application right
TA01 Transfer of patent application right

Effective date of registration: 20230307

Address after: Delaware USA

Applicant after: Benitec intellectual property Holding Co.,Ltd.

Address before: New South Wales Australia

Applicant before: Benetec Bio-Pharmaceuticals

GR01 Patent grant
GR01 Patent grant