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Paper Details

Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing.
Nat Commun
7
2023
ABE8, CRISPR prime-edited hepatocyte cell line, P281L, PAH, PAH variants, PKU, Phe, Phenylketonuria, adenine, autosomal recessive disorder, guide RNAs, humanized, mice, mouse, patients, phenylalanine, phenylalanine hydroxylase, phenylalanine hydroxylase (PAH) gene, phenylketonuria
Author NameAffiliation
Kiran MusunuruCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania
Kiran MusunuruPerelman School of Medicine at the University of Pennsylvania
Kiran MusunuruPerelman School of Medicine at the University of Pennsylvania
Kiran MusunuruCardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania
Kiran MusunuruPerelman School of Medicine at the University of Pennsylvania
Kiran MusunuruPerelman School of Medicine at the University of Pennsylvania
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