BR112021017054A2 - Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) - Google Patents

Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd)

Info

Publication number
BR112021017054A2
BR112021017054A2 BR112021017054A BR112021017054A BR112021017054A2 BR 112021017054 A2 BR112021017054 A2 BR 112021017054A2 BR 112021017054 A BR112021017054 A BR 112021017054A BR 112021017054 A BR112021017054 A BR 112021017054A BR 112021017054 A2 BR112021017054 A2 BR 112021017054A2
Authority
BR
Brazil
Prior art keywords
opmd
compositions
aav
methods
muscular dystrophy
Prior art date
Application number
BR112021017054A
Other languages
English (en)
Inventor
David Suhy
W Roelvink Petrus
Shih-Chu Kao
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 BR112021017054A2 publication Critical patent/BR112021017054A2/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14041Use of virus, viral particle or viral elements as a vector
    • C12N2710/14045Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14051Methods of production or purification of viral material
    • C12N2710/14052Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd).a presente divulgação se refere a vetores de entrega de vírus adenoassociado modificado (aav) compreendendo construções de dna "silenciar e substituir", composições compreendendo os mesmos, e o uso do aav modificado e composições para tratar a distrofia muscular oculofaríngea (opmd) em indivíduos que sofrem de opmd ou que sejam predispostos à mesma. em particular, a divulgação se refere ao aav contendo uma proteína de capsídeo com uma subunidade 1 modificada (vp1) e compreendendo uma construção de dna "silenciar e substituir", em que a construção de dna "silenciar e substituir" compreende (i) uma construção de rnai dirigida por dna (ddrnai) que codifica microrna curto em grampo (shmir) direcionado a pabpn1 causadora da opmd e (ii) uma construção de pabpn1 que codifica a proteína pabpn1 funcional tendo um transcrito de rnam que não é alvo dos shmirs em (i). a presente divulgação também se refere aos métodos para o tratamento da opmd que compreende a injeção direta de um aav da divulgação nos músculos faríngeos de um indivíduo.
BR112021017054A 2019-02-28 2020-02-28 Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd) BR112021017054A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962812187P 2019-02-28 2019-02-28
PCT/AU2020/050182 WO2020172720A1 (en) 2019-02-28 2020-02-28 Compositions and methods for treating oculopharyngeal muscular dystrophy (opmd)

Publications (1)

Publication Number Publication Date
BR112021017054A2 true BR112021017054A2 (pt) 2021-11-09

Family

ID=72238736

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021017054A BR112021017054A2 (pt) 2019-02-28 2020-02-28 Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd)

Country Status (12)

Country Link
US (1) US20220098614A1 (pt)
EP (1) EP3931317A4 (pt)
JP (1) JP2022522166A (pt)
KR (1) KR20210138030A (pt)
CN (1) CN113631706A (pt)
AU (1) AU2020229886A1 (pt)
BR (1) BR112021017054A2 (pt)
CA (1) CA3130221A1 (pt)
IL (1) IL285841A (pt)
MX (1) MX2021010412A (pt)
SG (1) SG11202108469XA (pt)
WO (1) WO2020172720A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020077412A1 (en) * 2018-10-17 2020-04-23 Benitec Biopharma Limited Methods for treating oculopharyngeal muscular dystrophy (opmd)

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
AU715543B2 (en) 1995-09-08 2000-02-03 Genzyme Corporation Improved AAV vectors for gene therapy
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
US8163543B2 (en) 2005-10-20 2012-04-24 Amsterdam Molecular Therapeutics B.V. AAV vectors produced in insect cells
WO2007120533A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
AU2007261806B2 (en) 2006-06-21 2013-08-15 Uniqure Ip B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells
AU2009209408B2 (en) 2008-01-29 2015-06-11 Beacon Therapeutics Limited Recombinant virus production using mammalian cells in suspension
KR20220106852A (ko) * 2013-07-26 2022-07-29 유니버시티 오브 아이오와 리써치 파운데이션 뇌 질환을 치료하기 위한 방법 및 조성물
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
CN104815338A (zh) * 2015-05-01 2015-08-05 许瑞安 一种携带kal基因重组腺相关病毒载体的基因治疗药物
WO2017184879A1 (en) 2016-04-21 2017-10-26 Virovek, Inc Aav production in insect cells, methods and compositions therefor
CN118421624A (zh) 2016-12-14 2024-08-02 贝尼泰克知识产权控股股份有限公司 用于治疗眼咽肌营养不良症(opmd)的试剂及其用途
AU2018323049A1 (en) 2017-08-31 2020-04-16 Benitec IP Holdings Inc. Adeno-associated virus (AAV) with modified phospholipase domain

Also Published As

Publication number Publication date
EP3931317A4 (en) 2023-01-04
CA3130221A1 (en) 2020-09-03
WO2020172720A1 (en) 2020-09-03
KR20210138030A (ko) 2021-11-18
MX2021010412A (es) 2022-01-24
AU2020229886A1 (en) 2021-09-09
EP3931317A1 (en) 2022-01-05
SG11202108469XA (en) 2021-09-29
IL285841A (en) 2021-10-31
US20220098614A1 (en) 2022-03-31
JP2022522166A (ja) 2022-04-14
CN113631706A (zh) 2021-11-09

Similar Documents

Publication Publication Date Title
Pothlichet et al. Cutting edge: innate immune response triggered by influenza A virus is negatively regulated by SOCS1 and SOCS3 through a RIG-I/IFNAR1-dependent pathway
ES2732023T3 (es) Supresión del gen de la huntingtina inducida por ARNi
Zhou et al. Honeysuckle-encoded atypical microRNA2911 directly targets influenza A viruses
US20160208255A1 (en) Rna agents for p21 gene modulation
Ben Khalifa et al. The matrix protein of rabies virus binds to RelAp43 to modulate NF-κB-dependent gene expression related to innate immunity
Santangelo et al. Effective reduction of the interleukin-1β transcript in osteoarthritis-prone guinea pig chondrocytes via short hairpin RNA mediated RNA interference influences gene expression of mediators implicated in disease pathogenesis
Yu et al. MiR-200c regulates ROS-induced apoptosis in murine BV-2 cells by targeting FAP-1
BR112021017054A2 (pt) Composições e métodos para o tratamento da distrofia muscular oculofaríngea (opmd)
JP2024105681A (ja) 筋強直性ジストロフィープロテインキナーゼの発現を抑制および/またはdmpk遺伝子の3’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
Zhu et al. Human TRA2A determines influenza A virus host adaptation by regulating viral mRNA splicing
JP2019515663A5 (pt)
Tarigan et al. Role of pattern recognition receptors and interferon-beta in protecting bat cell lines from encephalomyocarditis virus and Japanese encephalitis virus infection
Zhou et al. RNA interference of avian influenza virus H5N1 by inhibiting viral mRNA with siRNA expression plasmids
Xu et al. RNA interference of influenza A virus replication by microRNA-adapted lentiviral loop short hairpin RNA
Wang et al. Identification of miRNomes reveals ssc-miR-30d-R_1 as a potential therapeutic target for PRRS viral infection
JP2024119830A5 (pt)
CN102352355A (zh) 抑制Nav1.8的短干扰核酸组合物和方法
Zhu et al. Contribution of growth arrest-specific 5/miR-674 to the hypothalamus pituitary adrenal axis regulation effect by electroacupuncture following trauma
BR112019012312A8 (pt) Reagentes para tratamento da distrofia muscular oculofaríngea (opmd) e uso dos mesmos
JP2020503014A5 (pt)
Zhang et al. Nuclear transporter karyopherin subunit alpha 3 levels modulate Porcine circovirus type 2 replication in PK-15 cells
Al-Salleeh et al. TLR3 and TLR7 are involved in expression of IL-23 subunits while TLR3 but not TLR7 is involved in expression of IFN-β by Theiler's virus-infected RAW264. 7 cells
WO2018070510A1 (ja) 筋分化誘導剤
Sudaryatma et al. Bovine respiratory syncytial virus decreased pasteurella multocida adherence by downregulating the expression of intercellular adhesion molecule-1 on the surface of upper respiratory epithelial cells
Zhao et al. Small interfering RNA inhibits SARS-CoV nucleocapsid gene expression in cultured cells and mouse muscles