Development of an mRNA replacement therapy for phenylketonuria

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...

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Bibliographic Details
Published in:Molecular Therapy: Nucleic Acids
Main Authors: Carlos G. Perez-Garcia, Ramon Diaz-Trelles, Jerel Boyd Vega, Yanjie Bao, Marciano Sablad, Patty Limphong, Simon Chikamatsu, Hailong Yu, Wendy Taylor, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula
Format: Article
Language:English
Published: Elsevier 2022-06-01
Subjects:
MT: Delivery Strategies
mRNA
replacement
lipid nanoparticle
PKU
phenylketonuria
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122000488

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http://www.sciencedirect.com/science/article/pii/S2162253122000488

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