AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Richards, DY; Winn, SR; Dudley, S; Nygaard, S; Mighell, TL; Grompe, M; Harding, CO

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020; 17 (): 234

Abstract

Phenylketonuria (PKU) due to recessively inherited phenylalanine hydroxylase (PAH) deficiency results in hyperphenylalaninemia, which is toxic to the ......

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