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publicationDate 2017-07-19-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber EP-3191139-A1
titleOfInvention Rpgr gene therapy for retinitis pigmentosa
abstract Methods for treating a human subject who has X-linked Retinitis Pigmentosa (XLRP) or another clinically-defined ophthalmological condition due to a loss-of-function mutation in the gene encoding the retinitis pigmentosa GTPase regulator (RPGR) protein, the method comprising administering to the subject a nucleic acid comprising an adeno-associated viral vector comprising an abbreviated human RPGR cDNA.
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