Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Human Gene Therapy

Volumn 29, Issue 3, 2018, Pages 285-298

  Hinderer, Christian ^a   Katz, Nathan ^a   Buza, Elizabeth L ^a   Dyer, Cecilia ^a   Goode, Tamara ^a   Bell, Peter ^a   Richman, Laura K ^a   Wilson, James M ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b Institute for Human Gene Therapy   (United States)

Author keywords

adeno associated virus; axonopathy; gene therapy; hepatic toxicity; liver failure; shock

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; AMINOTRANSFERASE; SURVIVAL MOTOR NEURON PROTEIN; SMN1 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 1;

ACUTE LIVER FAILURE; ADENO ASSOCIATED VIRUS 9; ALPHA NERVE FIBER; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BODY WEIGHT; CONTROLLED STUDY; DRUG MEGADOSE; ENZYME LINKED IMMUNOSPOT ASSAY; HEMATOCRIT; HUMAN; IMMUNE RESPONSE; IN SITU HYBRIDIZATION; INTESTINE LYMPHATIC TISSUE; LIVER CELL; MOTONEURON; NONHUMAN; NUCLEOTIDE SEQUENCE; OBSERVATION; PROMOTER REGION; RHESUS MONKEY; SENSORY GANGLION; SENSORY NERVE CELL; SHOCK; SPINAL MUSCULAR ATROPHY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; YUCATAN MICROPIG; ANIMAL; BIOSYNTHESIS; BLOOD; DEPENDOPARVOVIRUS; GENE VECTOR; GENETICS; HAPLORHINI; METABOLISM; PATHOLOGY; PHARMACOLOGY; PIG; SPINAL GANGLION; TIME FACTOR; TRANSGENE;

ANIMALS; DEPENDOVIRUS; GANGLIA, SPINAL; GENETIC VECTORS; HAPLORHINI; HUMANS; SENSORY RECEPTOR CELLS; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SWINE; TIME FACTORS; TRANSAMINASES; TRANSGENES;

EID: 85043267068     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2018.015     Document Type: Article

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