WO1998048027A3 - Materials and methods for treatment of retinal diseases - Google Patents

Materials and methods for treatment of retinal diseases Download PDF

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Publication number
WO1998048027A3
WO1998048027A3 PCT/US1998/008003 US9808003W WO9848027A3 WO 1998048027 A3 WO1998048027 A3 WO 1998048027A3 US 9808003 W US9808003 W US 9808003W WO 9848027 A3 WO9848027 A3 WO 9848027A3
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WO
WIPO (PCT)
Prior art keywords
methods
materials
retinal diseases
treatment
retinal
Prior art date
Application number
PCT/US1998/008003
Other languages
French (fr)
Other versions
WO1998048027A9 (en
WO1998048027A2 (en
Inventor
William W Hauswirth
John G Flannery
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
Priority to BR9809284-7A priority Critical patent/BR9809284A/en
Priority to CA002287495A priority patent/CA2287495A1/en
Priority to JP54626898A priority patent/JP2001527399A/en
Priority to AU74675/98A priority patent/AU7467598A/en
Priority to EP98922042A priority patent/EP0977880A2/en
Publication of WO1998048027A2 publication Critical patent/WO1998048027A2/en
Publication of WO1998048027A3 publication Critical patent/WO1998048027A3/en
Publication of WO1998048027A9 publication Critical patent/WO1998048027A9/en

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • C12N2310/12—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

Abstract

The subject invention provides materials and methods for efficient, specific expression of proteins in retinal photoreceptor cells. Specifically, the constructs are composed of an adeno-associated vital vector containing a rod or cone-opsin promoter. These materials and methods can be used in therapies for retinal diseases. In one embodiment, ribozymes which degrade mutant mRNA are used to treat retinitis pigmentosa.
PCT/US1998/008003 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases WO1998048027A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
BR9809284-7A BR9809284A (en) 1997-04-21 1998-04-21 Construct and process for high-level polynucleotide expression
CA002287495A CA2287495A1 (en) 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases
JP54626898A JP2001527399A (en) 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases
AU74675/98A AU7467598A (en) 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases
EP98922042A EP0977880A2 (en) 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US4449297P 1997-04-21 1997-04-21
US4614697P 1997-05-09 1997-05-09
US60/044,492 1997-05-09
US60/046,146 1997-05-09

Publications (3)

Publication Number Publication Date
WO1998048027A2 WO1998048027A2 (en) 1998-10-29
WO1998048027A3 true WO1998048027A3 (en) 1999-03-25
WO1998048027A9 WO1998048027A9 (en) 1999-04-29

Family

ID=26721632

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/008003 WO1998048027A2 (en) 1997-04-21 1998-04-21 Materials and methods for treatment of retinal diseases

Country Status (6)

Country Link
EP (1) EP0977880A2 (en)
JP (1) JP2001527399A (en)
AU (1) AU7467598A (en)
BR (1) BR9809284A (en)
CA (1) CA2287495A1 (en)
WO (1) WO1998048027A2 (en)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040234999A1 (en) * 1996-04-02 2004-11-25 Farrar Gwenyth Jane Genetic suppression and replacement
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
WO1998048009A2 (en) * 1997-04-21 1998-10-29 University Of Florida Materials and methods for ribozyme treatment of retinal diseases
WO2000066780A2 (en) 1999-04-30 2000-11-09 University Of Florida Adeno-associated virus-delivered ribozyme compositions and methods of use
AU2001295193A1 (en) * 2000-05-01 2001-11-12 Novartis Ag Vectors for ocular transduction and use thereof for genetic therapy
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
EP2019588A4 (en) 2006-05-04 2010-11-24 Univ Wayne State Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP5852969B2 (en) 2010-02-26 2016-02-03 コーネル ユニヴァーシティー Artificial retina
WO2011127088A2 (en) 2010-04-05 2011-10-13 Eos Neuroscience, Inc. Methods and compositions for decreasing chronic pain
US9453241B2 (en) 2010-05-04 2016-09-27 Wayne State University AAV-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
CN103260560A (en) 2010-08-31 2013-08-21 康奈尔大学 Retina prosthesis
US9302103B1 (en) 2010-09-10 2016-04-05 Cornell University Neurological prosthesis
CN105349492B (en) 2011-05-18 2019-04-16 加利福尼亚大学董事会 For treating the composition and method of retinal disease
KR101976048B1 (en) 2011-08-25 2019-05-09 코넬 유니버시티 Retinal encoder for machine vision
WO2013063601A1 (en) * 2011-10-28 2013-05-02 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
HUE040487T2 (en) 2012-03-05 2019-03-28 Univ Wayne State Identification of channelrhodopsin-2 (chop2) mutations and methods of use
WO2016172188A1 (en) 2015-04-20 2016-10-27 Cornell University Machine vision with dimensional data reduction
CN113347962A (en) * 2019-01-23 2021-09-03 佛罗里达大学研究基金会有限公司 High efficiency transduction and lateral spread of novel AAV viruses in the retina enhanced by rational design

Citations (1)

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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

Patent Citations (1)

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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

Non-Patent Citations (12)

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Title
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CHEN J ET AL: "The human blue opsin promoter directs transgene expression in short-wave cones and bipolar cells in the mouse retina.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, (1994 MAR 29) 91 (7) 2611-5., XP002091808 *
CHIU M I ET AL: "A SEQUENCE UPSTREAM OF THE MOUSE BLUE VISUAL PIGMENT GENE DIRECTS BLUE CONE - SPECIFIC TRANSGENE EXPRESSION IN MOUSE RETINAS", VISUAL NEUROSCIENCE, (JUL/AUG 1994) VOL. 11, NO. 4, PP. 773-780., XP002091807 *
DAIGER, S. ET AL.: "Correlation of phenotype with genotype in inherited retinal degeneration.", BEHAVIORAL AND BRAIN SCIENCES, vol. 18, 1995, pages 452 - 467, XP002077238 *
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Also Published As

Publication number Publication date
WO1998048027A9 (en) 1999-04-29
CA2287495A1 (en) 1998-10-29
BR9809284A (en) 2004-08-03
WO1998048027A2 (en) 1998-10-29
AU7467598A (en) 1998-11-13
JP2001527399A (en) 2001-12-25
EP0977880A2 (en) 2000-02-09

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