Nuclease-mediated gene therapies for inherited metabolic diseases of the liver

Yale Journal of Biology and Medicine

Volumn 90, Issue 4, 2017, Pages 553-566

  Bryson, Taylor E ^a   Anglin, Caitlin M ^a   Bridges, P Hudson ^a   Cottle, Renee N ^a  

^a AMRL   (United States)

Author keywords

Gene therapy; Genome editing; Hepatocytes; Inborn errors of metabolism; Inherited metabolic disease of the liver; Nucleases; Therapy for rare disease

Indexed keywords

ALPHA 1 ANTITRYPSIN; BLOOD CLOTTING FACTOR 8; FUMARYLACETOACETASE; LOW DENSITY LIPOPROTEIN RECEPTOR; NUCLEASE; ORNITHINE CARBAMOYLTRANSFERASE; DEOXYRIBONUCLEASE;

ALPHA 1 ANTITRYPSIN DEFICIENCY; AUTOSOMAL RECESSIVE DISORDER; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CRISPR-CAS9 SYSTEM; FAMILIAL HYPERCHOLESTEROLEMIA; GENE EDITING; GENE SEQUENCE; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC ENGINEERING; HEMOPHILIA; HEMOPHILIA B; HEREDITARY TYROSINEMIA TYPE 1; HUMAN; LIVER DISEASE; METABOLIC LIVER DISEASE; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; POINT MUTATION; PROMOTER REGION; REVIEW; TRANSGENE; TYROSINEMIA; VIRAL GENE DELIVERY SYSTEM; DEPENDOPARVOVIRUS; GENETICS; HEMOPHILIA A; METABOLIC DISORDER; METABOLISM; MUTATION; PROCEDURES;

CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; DEOXYRIBONUCLEASES; DEPENDOVIRUS; GENE EDITING; GENETIC THERAPY; HEMOPHILIA A; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; LIVER DISEASES; METABOLIC DISEASES; MUTATION; ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE; TYROSINEMIAS;

EID: 85038588856     PISSN: 00440086     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

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