COMPOSITIONS AND METHODS FOR TREATMENT OF HEMOCHROMATOSIS

This application provides polynucleotides comprising a coding sequence for a functionally active hereditary hemochromatosis protein (HFE) or a functionally active fragment thereof. The invention further provides compositions comprising said polynucleotides and their use in methods of preventing or t...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: DAUGHERTY Sean Christopher, CARSON Rosaline Do, CATALDO Jason Robert, WONG Timothy Preston
Format: Patent
Sprache:eng ; spa
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Zusammenfassung:This application provides polynucleotides comprising a coding sequence for a functionally active hereditary hemochromatosis protein (HFE) or a functionally active fragment thereof. The invention further provides compositions comprising said polynucleotides and their use in methods of preventing or treating hemochromatosis in a subject. La presente solicitud proporciona polinucleótidos que comprenden una secuencia codificante para una proteína funcionalmente activa de hemocromatosis hereditaria (HFE) o un fragmento funcionalmente activo de la misma. La invención proporciona además composiciones que comprenden dichos polinucleótidos y su uso en métodos para prevenir o tratar la hemocromatosis en un sujeto.