Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 5053.

Details for Australian Patent Application No. 2009310558 (hide)

Owner Academisch Ziekenhuis Leiden Prosensa B.V. Prosensa Technologies B.V. Prosensa Holding BV

Inventors De Kimpe, Josephus Johannes; Platenburg, Gerard Johannes; Van Deutekom, Judith Christina Theodora; Aartsma-Rus, Annemieke; Van Ommen, Garrit-Jan Boudewijn

Agent Freehills

Pub. Number AU-A-2009310558

PCT Pub. Number WO2010/050802

Priority PCT/ NL2008/050673 27.10.08 NL

Filing date 11 March 2009

Wipo publication date 6 May 2010

International Classifications

A61K 31/7105 (2006.01) - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links

A61K 48/00 (2006.01) Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases

A61P 21/00 (2006.01) Drugs for disorders of the muscular or neuromuscular system

C12N 5/10 (2006.01) Undifferentiated human, animal or plant cells, e.g. cell lines - Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells

Event Publications

19 May 2011 PCT application entered the National Phase

  PCT publication WO2010/050802 Priority application(s): WO2010/050802

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