公开(公告)号：US20250000957A1

公开(公告)日：2025-01-02

申请号：US18690408

申请日：2022-09-15

Applicant: Arcturus Therapeutics, Inc.

Inventor： Ramon DIAZ TRELLES ,  Kiyoshi TACHIKAWA ,  Padmanabh CHIVUKULA

IPC: A61K38/51 ,  A61K48/00 ,  A61P3/00 ,  C12N9/88 ,  C12N15/88

Abstract: Phenylketonuria (PKU) is caused by a mutation in the phenylalanine hydroxylase (P AH) gene in the liver, Provided herein are compositions and methods for treating phenylketonuria (PKU) and related disorders. Polynucleotides for expressing bacterial and plant-derived phenylalanine ammonia lyase (PAL) are provided herein. Also provided herein are methods of treating PKU and related disorders that include administration of polynucleotides encoding PAL proteins.