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Application filed by Homology Medicines IncfiledCriticalHomology Medicines Inc
Publication of AR122165A1publicationCriticalpatent/AR122165A1/en
Medicines Containing Material From Animals Or Micro-Organisms
(AREA)
Abstract
En la presente se proporcionan composiciones de virus adenoasociados recombinantes (rAAV) que pueden restaurar la función del gen de la fenilalanina hidroxilasa (PAH) en las células, y métodos para el uso de estas para tratar enfermedades asociadas con la reducción de la función del gen PAH (por ejemplo, PKU). También se proporcionan ácidos nucleicos, vectores, sistemas de empaquetamiento, y métodos para fabricar las composiciones de virus adenoasociados.Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods of using these to treat diseases associated with reduced gene function. PAH (for example, PKU). Nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions are also provided.
ARP210101411A2020-05-272021-05-26
ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USING THESE
AR122165A1
(en)