WO2005016274A3 - Rnai treatment of eye disease - Google Patents

Rnai treatment of eye disease Download PDF

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Publication number
WO2005016274A3
WO2005016274A3 PCT/US2004/025276 US2004025276W WO2005016274A3 WO 2005016274 A3 WO2005016274 A3 WO 2005016274A3 US 2004025276 W US2004025276 W US 2004025276W WO 2005016274 A3 WO2005016274 A3 WO 2005016274A3
Authority
WO
WIPO (PCT)
Prior art keywords
eye disease
expression
present
rnai
tshr
Prior art date
Application number
PCT/US2004/025276
Other languages
French (fr)
Other versions
WO2005016274A2 (en
Inventor
Maya Eibschitz-Tsimhoni
Original Assignee
Univ The Michigan
Maya Eibschitz-Tsimhoni
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 The Michigan, Maya Eibschitz-Tsimhoni filed Critical Univ The Michigan
Publication of WO2005016274A2 publication Critical patent/WO2005016274A2/en
Publication of WO2005016274A3 publication Critical patent/WO2005016274A3/en

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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
    • C12N15/1138—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
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention relates to RNA interference (RNAi) for preventing or reducing the expression of the thyroid stimulating hormone receptor gene in a target cell. In particular, the present invention provides compositions comprising small interfering RNA duplexes (siRNAs), or vectors that encode SIRNA, that inhibit the expression of the TSHr gene (e.g. by targeting TSHr mRNA), and methods of using these compositions to treat or prevent eye disease (e.g. Graves’ ophthalmopathy) in a patient.
PCT/US2004/025276 2003-08-07 2004-08-05 Rnai treatment of eye disease WO2005016274A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US49315603P 2003-08-07 2003-08-07
US60/493,156 2003-08-07

Publications (2)

Publication Number Publication Date
WO2005016274A2 WO2005016274A2 (en) 2005-02-24
WO2005016274A3 true WO2005016274A3 (en) 2005-07-14

Family

ID=34193168

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/025276 WO2005016274A2 (en) 2003-08-07 2004-08-05 Rnai treatment of eye disease

Country Status (1)

Country Link
WO (1) WO2005016274A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5998148A (en) * 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
US20030021780A1 (en) * 2000-10-06 2003-01-30 Smith Terry J. Diagnosis and therapy of antibody-mediated inflammatory autoimmune disorders
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5998148A (en) * 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
US20030021780A1 (en) * 2000-10-06 2003-01-30 Smith Terry J. Diagnosis and therapy of antibody-mediated inflammatory autoimmune disorders
US20030143204A1 (en) * 2001-07-27 2003-07-31 Lewis David L. Inhibition of RNA function by delivery of inhibitors to animal cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SANDERS ET AL.: "Human monoclonal thyroid stimulating autoantibody", THE LANCET, vol. 362, July 2003 (2003-07-01), pages 126 - 128, XP004783432 *

Also Published As

Publication number Publication date
WO2005016274A2 (en) 2005-02-24

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