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BR112017022621A2 - liberação do gene de smad7 como uma substância terapêutica - Google Patents

liberação do gene de smad7 como uma substância terapêutica

Info

Publication number
BR112017022621A2
BR112017022621A2 BR112017022621A BR112017022621A BR112017022621A2 BR 112017022621 A2 BR112017022621 A2 BR 112017022621A2 BR 112017022621 A BR112017022621 A BR 112017022621A BR 112017022621 A BR112017022621 A BR 112017022621A BR 112017022621 A2 BR112017022621 A2 BR 112017022621A2
Authority
BR
Brazil
Prior art keywords
therapeutic substance
vectors
smad7 gene
gene release
aav
Prior art date
Application number
BR112017022621A
Other languages
English (en)
Inventor
Dantese Rodgers Buel
Gregorevic Paul
Original Assignee
Baker Idi Heart And Diabetes Inst
Univ Washington State
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 Baker Idi Heart And Diabetes Inst, Univ Washington State filed Critical Baker Idi Heart And Diabetes Inst
Publication of BR112017022621A2 publication Critical patent/BR112017022621A2/pt

Links

Classifications

    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

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

Abstract

são descritos neste documento vetores, tais como os veto-res de vírus adeno-associado (aav), e aav recombinante (raav) ex-pressando a smad7. os vetores de aav e o raav divulgados podem ser usados para aplicações terapêuticas no tratamento e na atenuação da perda muscular, da perda dos músculos cardíacos e/ou esqueléticos associada com a caquexia por câncer.
BR112017022621A 2015-04-23 2016-04-22 liberação do gene de smad7 como uma substância terapêutica BR112017022621A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562151547P 2015-04-23 2015-04-23
PCT/US2016/029018 WO2016172608A1 (en) 2015-04-23 2016-04-22 Smad7 gene delivery as a therapeutic

Publications (1)

Publication Number Publication Date
BR112017022621A2 true BR112017022621A2 (pt) 2018-07-17

Family

ID=57143535

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017022621A BR112017022621A2 (pt) 2015-04-23 2016-04-22 liberação do gene de smad7 como uma substância terapêutica

Country Status (11)

Country Link
US (1) US11268107B2 (pt)
EP (1) EP3285813B1 (pt)
JP (1) JP2018519250A (pt)
KR (1) KR20180002706A (pt)
CN (1) CN107847612A (pt)
AU (1) AU2016252887B2 (pt)
BR (1) BR112017022621A2 (pt)
CA (1) CA2983808C (pt)
ES (1) ES2856936T3 (pt)
PT (1) PT3285813T (pt)
WO (1) WO2016172608A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108753943A (zh) * 2018-01-17 2018-11-06 中国医学科学院阜外医院 miR-216a及其靶基因在血管衰老和动脉粥样硬化性心脏病中的应用
CN108498798A (zh) * 2018-04-17 2018-09-07 上海市第六人民医院 一种防治骨骼肌萎缩的治疗药物靶点及其应用
EP4135753A4 (en) 2020-04-17 2024-05-01 The Trustees of Columbia University in the City of New York METHODS, COMPOSITIONS AND THEIR USES FOR REVERSING SARCOPENIA

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR042545A1 (es) 2002-12-20 2005-06-22 Amgen Inc Agentes ligantes que inhiben la miostatina
SI2277996T1 (sl) 2003-05-21 2014-12-31 Genzyme Corporation Postopki za izdelavo pripravkov rekombinantnih AAV virionov v bistvu brez praznih kapsid
US7655467B2 (en) 2003-11-14 2010-02-02 The University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
CA2787827C (en) 2010-01-28 2020-11-10 The Children's Hospital Of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US9084746B2 (en) 2010-09-22 2015-07-21 The Regents Of The University Of Colorado, A Body Corporate Therapeutic applications of SMAD7
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
US20140127228A1 (en) * 2012-11-02 2014-05-08 Indiana University School of Medicine Inhibition of tgfbeta signaling to improve muscle function in cancer
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective

Also Published As

Publication number Publication date
CA2983808A1 (en) 2016-10-27
EP3285813A1 (en) 2018-02-28
AU2016252887B2 (en) 2021-08-12
CN107847612A (zh) 2018-03-27
JP2018519250A (ja) 2018-07-19
AU2016252887A1 (en) 2017-10-26
PT3285813T (pt) 2021-03-05
WO2016172608A1 (en) 2016-10-27
ES2856936T3 (es) 2021-09-28
KR20180002706A (ko) 2018-01-08
EP3285813B1 (en) 2020-12-23
CA2983808C (en) 2022-03-22
EP3285813A4 (en) 2018-10-10
US11268107B2 (en) 2022-03-08
US20180112232A1 (en) 2018-04-26

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Legal Events

Date Code Title Description
B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]