Development of an mRNA replacement therapy for phenylketonuria

• Main Authors: Carlos G. Perez-Garcia (Author), Ramon Diaz-Trelles (Author), Jerel Boyd Vega (Author), Yanjie Bao (Author), Marciano Sablad (Author), Patty Limphong (Author), Simon Chikamatsu (Author), Hailong Yu (Author), Wendy Taylor (Author), Priya P. Karmali (Author), Kiyoshi Tachikawa (Author), Padmanabh Chivukula (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Phenylketonuria (PKU) is an inborn error caused by deficiencies in phenylalanine (Phe) metabolism. Mutations in the phenylalanine hydroxylase (PAH) gene are the main cause of the disease whose signature hallmarks of toxically elevated levels of Phe accumulation in plasma and organs such as the brain...