Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
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United States of America Patent
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Feb 9, 2016
Grant Date -
Aug 8, 2013
app pub date -
Jul 29, 2009
filing date -
Jul 29, 2008
priority date (Note) -
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Abstract
Methods and compositions for treating retinal diseases comprising therapeutic amounts of a compound selected from a normal Puf-A gene product, an active polypeptide fragment thereof, an analog thereof or a peptidomimetic thereof. Vectors, including AAV vectors comprising the therapeutic compound are provided. Puf-A compositions suitable for subretinal, intravitreal, topical, subconjunctival, retrobulbar, periocular, suprachoroidal, or intraocular administration are provided. Methods for screening siRNA, RNAi and shRNA, small molecules and monoclonal antibodies that inhibit Puf-A target activity and reduce apoptosis are provided.
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