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US 2023/0414724 A1Published application
Treatment of hyperbilirubinemia
AssigneesGenethon, International Centre for Genetic Engineering and Biotechnology
InventorsFEDERICO MINGOZZI, GIUSEPPE RONZITTI, FANNY COLLAUD, ANDRÉS MURO, GIULIA BORTOLUSSI
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Abstract
The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.
Claims13
An intron which is a modified intron with decreased open reading frames.
Dependencies are inferred from the claim text.