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Open AccessRapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing
Phenylketonuria (PKU), an autosomal recessive disorder caused by pathogenic variants in the phenylalanine hydroxylase (PAH) gene, results in the accumulation of blood phenylalanine (Phe) to neurotoxic levels. Cur...
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Open AccessHallmarks of primary neurulation are conserved in the zebrafish forebrain
Primary neurulation is the process by which the neural tube, the central nervous system precursor, is formed from the neural plate. Incomplete neural tube closure occurs frequently, yet underlying causes remai...
