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US 2018/0273594 A1Published application
Treatment of retinitis pigmentosa
OwnerRobert MACLAREN, Dominik FISCHERinventors
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Abstract
A polynucleotide comprising a nucleotide sequence encoding the retinitis pigmentosa GTPase regulator ORF15 isoform (RPGRORF15), wherein the RPGRORF15-encoding nucleotide sequence has been codon optimised to increase fidelity of replication of the sequence.
Claims12
1-48. (canceled)
A composition comprising a nucleic acid comprising
- (a) a sequence encoding a rhodopsin kinase promoter, and
- (b) a sequence encoding a retinitis pigmentosa GTPase regulator ORF15 isoform (RPGRORF15).
Dependencies are inferred from the claim text.