Targeting Metabolic Disorders: CRISPR/Cas9 Gene Therapy for Phenylketonuria
CRISPR/Cas9 gene therapy offers promising prospects for treating phenylketonuria (PKU), a metabolic disorder caused by a phenylalanine hydroxylase (PAH) gene mutation. By precisely editing the PAH gene, CRISPR/Cas9 aims to restore PAH function, potentially alleviating the severe symptoms associated with PKU.



