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filingDate 2010-04-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2013-05-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_113baf0e47a0192c3d91d0ab04b3378c
publicationDate 2013-05-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-8450473-B2
titleOfInvention Compositions and methods for therapy of macular degeneration
abstract Provided are compositions and methods for therapy of macular degeneration including dry age-related macular degeneration (dAMD), juvenile macular degenerations (JMDs) where toxic retinoids are known to accumulate as part of the pathogenesis, such as Stargardt disease, and Best disease, and neovascular wet age-related macular degeneration. The method entails administering to an individual in need of therapy for macular degeneration a first polynucleotide that can facilitate a reduction in the amount of rod opsin (RHO) mRNA in the individual; or a second polynucleotide that can facilitate a reduction in the amount of RPE65 mRNA in the individual; or a combination thereof. The polynucleotides of the invention are hammerhead ribozymes or shRNAs. The polynucleotides target a sequence in RHO mRNA or RPE65 mRNA and facilitate reduction in the target mRNA via ribozymatic cleavage of the target, or by hybridization to the target, which leads to RNAi mediated degradation of the target mRNA.
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