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US 2023/0265456 A1Published application
Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
AssigneeFundacióN Para La InvestigacióN MéDica Aplicada
InventorsRuben HERNANDEZ ALCOCEBA, Ana RICOBARAZA ABARQUERO, Gloria GONZALEZ ASEGUINOLAZA
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Abstract
The present disclosure relates to gene therapy vector for use in the treatment of cerebro tendinous xanthomatosis. More specifically, the present invention relates to a nucleic acid construct comprising liver specific promoter operably linked to a nucleic acid sequence encoding for the sterol 27-hydroxylase for the treatment of CTX.
Claims20
A nucleic acid construct comprising a liver-specific promoter operably linked to a transgene encoding human sterol 27-hydroxylase or a functional variant thereof.
(canceled)
Dependencies are inferred from the claim text.