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Journal Article
WHO Drug Information, ISSN 1010-9609, 04/2018, Volume 32, Issue 2, pp. 215 - 215
Journal Article
Journal Article
Nature Medicine, ISSN 1078-8956, 10/2018, Volume 24, Issue 10, pp. 1519 - 1525
CRISPR-Cas-based genome editing holds great promise for targeting genetic disorders, including inborn errors of hepatocyte metabolism. Precise correction of... 
MEDICINE, RESEARCH & EXPERIMENTAL | STEM-CELLS | DNA CLEAVAGE | CRYSTAL-STRUCTURE | HUMAN-CELLS | BIOCHEMISTRY & MOLECULAR BIOLOGY | PHENYLKETONURIA | GUIDE RNA | CELL BIOLOGY | STAPHYLOCOCCUS-AUREUS CAS9 | GENE-THERAPY | HOMOLOGOUS RECOMBINATION | ALTERED PAM SPECIFICITIES | Phenylalanine Hydroxylase - genetics | Dependovirus - genetics | Liver - pathology | Liver Diseases - genetics | Phenylketonurias - genetics | Recombinational DNA Repair - genetics | Humans | Liver - metabolism | Liver Diseases - pathology | Liver Diseases - therapy | DNA - therapeutic use | CRISPR-Cas Systems - genetics | Gene Editing | DNA - genetics | Phenylketonurias - therapy | Animals | Phenylalanine Hydroxylase - therapeutic use | Phenylalanine - blood | Phenylketonurias - metabolism | Mice | Liver Diseases - metabolism | Phenylketonurias - pathology | Disease Models, Animal | Genetic Therapy - methods | Metabolic diseases | Care and treatment | Genetic aspects | Liver diseases | Gene therapy | Methods | Intravenous administration | Inborn errors of metabolism | Enzyme activity | Phenylalanine 4-monooxygenase | Liver | DNA damage | Editing | Viruses | Homology | Genomes | mRNA | DNA repair | Proteins | Reversion | Enzymatic activity | Restoration | Cargo capacity | Fusion protein | Repair | Deoxyribonucleic acid--DNA | CRISPR | Phenotypes | Phenylketonuria | Phenylalanine | Base pairs | Hydroxylase | Medical treatment | Metabolism | Mutation
Journal Article