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WO2005002498A3 - Methods of treating disorders caused by formation of transcripts carrying nonsense mutations - Google Patents

Methods of treating disorders caused by formation of transcripts carrying nonsense mutations Download PDF

Info

Publication number
WO2005002498A3
WO2005002498A3 PCT/IL2004/000556 IL2004000556W WO2005002498A3 WO 2005002498 A3 WO2005002498 A3 WO 2005002498A3 IL 2004000556 W IL2004000556 W IL 2004000556W WO 2005002498 A3 WO2005002498 A3 WO 2005002498A3
Authority
WO
WIPO (PCT)
Prior art keywords
formation
methods
treating disorders
disorders caused
nonsense mutations
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
PCT/IL2004/000556
Other languages
French (fr)
Other versions
WO2005002498A2 (en
Inventor
Batsheva Kerem
Liat Shushi
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.)
Yissum Research Development Co of Hebrew University of Jerusalem
Original Assignee
Yissum Research Development Co of Hebrew University of Jerusalem
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 Yissum Research Development Co of Hebrew University of Jerusalem filed Critical Yissum Research Development Co of Hebrew University of Jerusalem
Publication of WO2005002498A2 publication Critical patent/WO2005002498A2/en
Anticipated expiration legal-status Critical
Publication of WO2005002498A3 publication Critical patent/WO2005002498A3/en
Ceased legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

A method of treating an individual having a disorder caused by formation of an RNA transcript carrying a nonsense mutation is provided. The method is effected by exposing cells of the individual expressing the RNA transcript to treatment selected capable of increasing a half life of the RNA transcript in the cells; and exposing the cells to an aminoglycoside thereby treating the individual having the genetic disease caused by formation of the RNA transcript carrying the nonsense mutation.
PCT/IL2004/000556 2003-07-01 2004-06-22 Methods of treating disorders caused by formation of transcripts carrying nonsense mutations Ceased WO2005002498A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US48364103P 2003-07-01 2003-07-01
US60/483,641 2003-07-01

Publications (2)

Publication Number Publication Date
WO2005002498A2 WO2005002498A2 (en) 2005-01-13
WO2005002498A3 true WO2005002498A3 (en) 2006-02-02

Family

ID=33563945

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IL2004/000556 Ceased WO2005002498A2 (en) 2003-07-01 2004-06-22 Methods of treating disorders caused by formation of transcripts carrying nonsense mutations

Country Status (1)

Country Link
WO (1) WO2005002498A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2390255B1 (en) 2006-04-03 2016-07-13 Technion Research & Development Foundation Ltd. Novel aminoglycosides and uses thereof in the treatment of genetic disorders

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5827543A (en) * 1995-12-07 1998-10-27 The Procter & Gamble Company Methods and compositions for the prevention and treatment of urogenital disorders
US6475993B2 (en) * 1998-12-22 2002-11-05 Universite Laval Treatment of hereditary diseases with gentamicin

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5827543A (en) * 1995-12-07 1998-10-27 The Procter & Gamble Company Methods and compositions for the prevention and treatment of urogenital disorders
US6475993B2 (en) * 1998-12-22 2002-11-05 Universite Laval Treatment of hereditary diseases with gentamicin

Also Published As

Publication number Publication date
WO2005002498A2 (en) 2005-01-13

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