Adeno-associated viral vectors for correction of inborn errors of metabolism: Progressing towards clinical application

Current Pharmaceutical Design

Volumn 17, Issue 24, 2011, Pages 2500-2515

  Jacobs, Frank ^a,b   Wang, Lili ^b  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

AAV; Adeno associated viral vectors; Gene therapy; Liver; Metabolic diseases

Indexed keywords

ALPHA GALACTOSIDASE; N ACETYLGALACTOSAMINE 4 SULFATASE; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; PARVOVIRUS VECTOR 2; PARVOVIRUS VECTOR 6; PARVOVIRUS VECTOR 8; UNCLASSIFIED DRUG; VIRUS CAPSID ANTIGEN;

ACUTE HEART INFARCTION; ADAPTIVE IMMUNITY; AMINO ACID METABOLISM; CD8+ T LYMPHOCYTE; CELL FUNCTION; CYTOTOXIC T LYMPHOCYTE; DISEASE MODEL; FABRY DISEASE; FAMILIAL HYPERCHOLESTEROLEMIA; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSCRIPTION AND TRANSLATION; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 2; HEMOPHILIA B; HUMAN; INBORN ERROR OF METABOLISM; ISCHEMIC HEART DISEASE; LIVER CELL; LIVER TOXICITY; MAROTEAUX LAMY SYNDROME; METHYLMALONIC ACIDURIA; MUCOPOLYSACCHARIDOSIS; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; PEDIATRICS; PHASE 1 CLINICAL TRIAL (TOPIC); PHENYLKETONURIA; PRIORITY JOURNAL; PROPIONIC ACIDEMIA; REVIEW; SEROTYPE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; LIVER; METABOLISM, INBORN ERRORS; TRANSGENES;

EID: 80053526511     PISSN: 13816128     EISSN: 18734286     Source Type: Journal    
DOI: 10.2174/138161211797247569     Document Type: Review

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