BR9808606A - Materiais e métodos para tratamento de doenças com ribozimas - Google Patents

Materiais e métodos para tratamento de doenças com ribozimas

Info

Publication number
BR9808606A
BR9808606A BR9808606-5A BR9808606A BR9808606A BR 9808606 A BR9808606 A BR 9808606A BR 9808606 A BR9808606 A BR 9808606A BR 9808606 A BR9808606 A BR 9808606A
Authority
BR
Brazil
Prior art keywords
methods
materials
ribozymes
treating diseases
mrna
Prior art date
Application number
BR9808606-5A
Other languages
English (en)
Inventor
Alfred S Lewin
William W Haustwirth
Kimberly Drenser
Original Assignee
Univ Florida
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 Univ Florida filed Critical Univ Florida
Publication of BR9808606A publication Critical patent/BR9808606A/pt

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    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/127—DNAzymes
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Endocrinology (AREA)
  • Veterinary Medicine (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Ophthalmology & Optometry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

<B>MATERIAIS E MéTODOS PARA TRATAMENTO DE DOENçAS COM RIBOZIMAS<D>. A presente invenção provê materiais e métodos para a redução ou eliminação eficientes, específicas de mRNA indesejado. Esses materiais e métodos podem ser usados em terapias de doenças retinianas. Em uma modalidade, as ribozimas que degradam mRNA mutante são usadas para tratar retinite pigmentosa.
BR9808606-5A 1997-04-21 1998-04-21 Materiais e métodos para tratamento de doenças com ribozimas BR9808606A (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4449297P 1997-04-21 1997-04-21
US4614797P 1997-05-09 1997-05-09
PCT/US1998/007968 WO1998048009A2 (en) 1997-04-21 1998-04-21 Materials and methods for ribozyme treatment of retinal diseases

Publications (1)

Publication Number Publication Date
BR9808606A true BR9808606A (pt) 2000-05-23

Family

ID=26721633

Family Applications (1)

Application Number Title Priority Date Filing Date
BR9808606-5A BR9808606A (pt) 1997-04-21 1998-04-21 Materiais e métodos para tratamento de doenças com ribozimas

Country Status (8)

Country Link
US (1) US6225291B1 (pt)
EP (1) EP0977841A2 (pt)
JP (1) JP2001523959A (pt)
AU (1) AU735788B2 (pt)
BR (1) BR9808606A (pt)
CA (1) CA2287508A1 (pt)
NZ (1) NZ500427A (pt)
WO (1) WO1998048009A2 (pt)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2002542805A (ja) * 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ アデノ随伴ウイルス送達リボザイム組成物および使用方法
WO2000066720A2 (en) * 1999-04-30 2000-11-09 City Of Hope The use of adeno-associated virus (aav) deliver cytoprotective genes
US7829693B2 (en) * 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US20030003582A1 (en) * 2001-05-08 2003-01-02 Tranzyme, Inc. Trans-viral vector mediated gene transfer to the retina
AU2003222820A1 (en) 2002-04-18 2003-10-27 Acuity Pharmaceuticals, Inc. Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
AU2003290586B2 (en) * 2002-11-04 2009-07-02 University Of Massachusetts Allele-specific RNA interference
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
EP2140004B1 (en) 2007-04-12 2022-01-12 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Genetic suppression and replacement
AU2009322279A1 (en) 2008-12-04 2011-07-14 Opko Pharmaceuticals, Llc Compositions and methods for selective inhibition of pro-angiogenic VEGF isoforms
WO2010127209A2 (en) * 2009-04-30 2010-11-04 The Research Foundation Of State University Of New York Compositions and methods for therapy of macular degeneration
ES2911185T3 (es) 2010-04-23 2022-05-18 Univ Florida Composiciones de rAAV-guanilato ciclasa y métodos para tratar la amaurosis congénita de Leber 1 (LCA1)
CN114231563A (zh) 2014-05-02 2022-03-25 建新公司 用于视网膜及cns基因疗法的aav载体
EP4012035A1 (en) 2014-09-16 2022-06-15 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
WO2016044478A1 (en) 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP3261643B1 (en) 2015-02-26 2020-11-25 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin
US11653636B2 (en) * 2019-11-15 2023-05-23 University Of Ulsan Foundation For Industry Cooperation Method of making a rat model of retinal degeneration and rat model made thereby
AU2021213786A1 (en) 2020-01-29 2022-09-22 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US5037746A (en) 1986-12-03 1991-08-06 University Patents, Inc. RNA ribozyme polymerases, and methods
US5646020A (en) 1992-05-14 1997-07-08 Ribozyme Pharmaceuticals, Inc. Hammerhead ribozymes for preferred targets
US5498539A (en) 1992-07-02 1996-03-12 Daiichi Pharmaceutical Co., Ltd. Bovine endothelial nitric oxide synthase nucleic acids
US5639655A (en) 1993-01-19 1997-06-17 Ribozyme Pharmaceuticals, Inc. PML-RARA targeted ribozymes
US6410322B1 (en) * 1993-07-27 2002-06-25 Hybridon Inc Antisense oligonucleotide inhibition of vascular endothelial growth factor expression
US5646031A (en) 1995-05-16 1997-07-08 Northern Illinois University SArMV and sCYMVI hairpin ribozymes
GB9519299D0 (en) * 1995-09-21 1995-11-22 Farrar Gwyneth J Genetic strategy
GB9604449D0 (en) * 1996-03-01 1996-05-01 Farrar Gwyneth J Genetic strategy ii
BR9809284A (pt) * 1997-04-21 2004-08-03 Univ Florida Construto e processo para expressão de polinucleotìdeo em altos nìveis
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases

Also Published As

Publication number Publication date
EP0977841A2 (en) 2000-02-09
CA2287508A1 (en) 1998-10-29
JP2001523959A (ja) 2001-11-27
WO1998048009A2 (en) 1998-10-29
AU7140698A (en) 1998-11-13
NZ500427A (en) 2002-12-20
AU735788B2 (en) 2001-07-12
US6225291B1 (en) 2001-05-01
WO1998048009A3 (en) 1999-03-25

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Legal Events

Date Code Title Description
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]
B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO PUBLICADO NA RPI 1937 DE 19/02/2008