-
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
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.