METHODS AND COMPOSITIONS FOR EXPRESSING PHENYLALANINE HYDROXYLASE

The present disclosure provides expression constructs comprising a phenylalanine hydroxylase (PAH) transgene and methods of using the constructs for treating phenylketonuria (PKU).

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Bibliographische Detailangaben
1. Verfasser: de Alencastro, Gustavo
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present disclosure provides expression constructs comprising a phenylalanine hydroxylase (PAH) transgene and methods of using the constructs for treating phenylketonuria (PKU).