HUMAN PAH EXPRESSION CASSETTE FOR TREATMENT OF PKU BY LIVER-DIRECTED GENE REPLACEMENT THERAPY

Abstract: provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes a pah polypeptide. also provided are methods to treat phenylketonuria (pku) and/or to reduce levels of phenylalanine in an individual in need thereof. further provided herein are vectors (e.g., raav vectors), viral particles, pharmaceutical compositions and kits for expressing a pah polypeptide in an individual in need thereof.

Inventor(s): Sirkka R.M. KYOSTIO-MOORE

CPC Classification: C12N15/86 (MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media ))

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