AU735788B2 - Materials and methods for ribozyme treatment of diseases - Google Patents

Materials and methods for ribozyme treatment of diseases Download PDF

Info

Publication number
AU735788B2
AU735788B2 AU71406/98A AU7140698A AU735788B2 AU 735788 B2 AU735788 B2 AU 735788B2 AU 71406/98 A AU71406/98 A AU 71406/98A AU 7140698 A AU7140698 A AU 7140698A AU 735788 B2 AU735788 B2 AU 735788B2
Authority
AU
Australia
Prior art keywords
ribozyme
composition according
retinal
ribozymes
mutant
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.)
Ceased
Application number
AU71406/98A
Other languages
English (en)
Other versions
AU7140698A (en
Inventor
Kimberly Drenser
William W. Hauswirth
Alfred S. Lewin
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.)
University of Florida
Original Assignee
University of 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 University of Florida filed Critical University of Florida
Publication of AU7140698A publication Critical patent/AU7140698A/en
Application granted granted Critical
Publication of AU735788B2 publication Critical patent/AU735788B2/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • C12N15/11—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)
  • Chemical Kinetics & Catalysis (AREA)
  • Endocrinology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
AU71406/98A 1997-04-21 1998-04-21 Materials and methods for ribozyme treatment of diseases Ceased AU735788B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US4449297P 1997-04-21 1997-04-21
US60/044492 1997-04-21
US4614797P 1997-05-09 1997-05-09
US60/046147 1997-05-09
PCT/US1998/007968 WO1998048009A2 (en) 1997-04-21 1998-04-21 Materials and methods for ribozyme treatment of retinal diseases

Publications (2)

Publication Number Publication Date
AU7140698A AU7140698A (en) 1998-11-13
AU735788B2 true AU735788B2 (en) 2001-07-12

Family

ID=26721633

Family Applications (1)

Application Number Title Priority Date Filing Date
AU71406/98A Ceased AU735788B2 (en) 1997-04-21 1998-04-21 Materials and methods for ribozyme treatment of diseases

Country Status (8)

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

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ500427A (en) 1997-04-21 2002-12-20 Univ Florida The use of ribozyme treatment for 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
US20050222061A1 (en) 2002-04-18 2005-10-06 Schulte Ralf W Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
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
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
US8257969B2 (en) * 2007-04-12 2012-09-04 The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
JP5832293B2 (ja) 2008-12-04 2015-12-16 オプコ ファーマシューティカルズ、エルエルシー 血管新生促進ｖｅｇｆイソ型を選択的に抑制する組成物および方法
US8450473B2 (en) 2009-04-30 2013-05-28 The Research Foundation Of State University Of New York Compositions and methods for therapy of macular degeneration
HUE041571T2 (hu) 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
PT4012035T (pt) 2014-09-16 2025-02-11 Genzyme Corp Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc)
EP4012035B1 (en) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
MX391217B (es) 2015-02-26 2025-03-21 Ionis Pharmaceuticals Inc Moduladores de rodopsina p23h con especificidad de alelo.
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
WO2021155137A1 (en) 2020-01-29 2021-08-05 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997011169A2 (en) * 1995-09-21 1997-03-27 Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
AU7467598A (en) * 1997-04-21 1998-11-13 University Of Florida Materials and methods for treatment of retinal diseases

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5037746A (en) 1986-12-03 1991-08-06 University Patents, Inc. RNA ribozyme polymerases, and methods
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases 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
GB9604449D0 (en) 1996-03-01 1996-05-01 Farrar Gwyneth J Genetic strategy ii
NZ500427A (en) 1997-04-21 2002-12-20 Univ Florida The use of ribozyme treatment for retinal diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997011169A2 (en) * 1995-09-21 1997-03-27 Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
AU7467598A (en) * 1997-04-21 1998-11-13 University Of Florida Materials and methods for treatment of retinal diseases

Also Published As

Publication number Publication date
JP2001523959A (ja) 2001-11-27
BR9808606A (pt) 2000-05-23
WO1998048009A3 (en) 1999-03-25
CA2287508A1 (en) 1998-10-29
US6225291B1 (en) 2001-05-01
EP0977841A2 (en) 2000-02-09
WO1998048009A2 (en) 1998-10-29
AU7140698A (en) 1998-11-13
NZ500427A (en) 2002-12-20

Similar Documents

Publication Publication Date Title
US6225291B1 (en) Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
KR102670852B1 (ko) 변이형 RNAi
US7342111B2 (en) Adeno-associated virus-delivered ribozyme compositions and methods of use
KR101984145B1 (ko) 레베르 선천성 흑내장-1(lca-1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
AU7467598A (en) Materials and methods for treatment of retinal diseases
US9255130B2 (en) Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
Gorbatyuk et al. Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery
WO2008154482A2 (en) Sirna compositions and methods of use in treatment of ocular diseases
EA033653B1 (ru) Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера
JP2021534814A (ja) アルファ−シヌクレインに対するバリアントＲＮＡｉ
Gorbatyuk et al. Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach
Hauswirth et al. Ribozyme uses in retinal gene therapy
WO2002088320A2 (en) rAAV-VECTORED RIBOZYME COMPOSITIONS AND METHODS FOR THE TREATMENT OF RETINAL DISEASES
RU2763319C2 (ru) Реагенты для лечения окулофарингеальной мышечной дистрофии (офмд) и их применение
US20100041741A1 (en) Suppression of mitochondrial oxidative stress
CN112839682B (zh) 用于治疗卵黄状黄斑变性的腺相关病毒载体
CN114555084A (zh) 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法
Flannery et al. Ribozyme-mediated gene therapy for autosomal dominant retinal degeneration
US20090068147A1 (en) Raav vector-based pro-opiomelanocortin compositions and methods of use
Liu et al. Ribozyme knockdown of the γ-subunit of rod cGMP phosphodiesterase alters the ERG and retinal morphology in wild-type mice
CA2659353C (en) Composition and method for treatment of tumors
EA046920B1 (ru) Антисмысловые олигонуклеотиды, восстанавливающие аберрантный сплайсинг abca4
Nishikawa et al. John G. Flannery, Alfred S. Lewin, 24 Kimberly A. Drenser, 2
Hauswirth et al. Viral-vectored ribozymes as therapy for autosomal dominant retinal disease
AU2002256409A1 (en) Raav-vectored ribozyme compositions and methods for the treatment of rentinal diseases

Legal Events

Date Code Title Description
SREP Specification republished
FGA Letters patent sealed or granted (standard patent)