Adeno-Associated Virus Gene Therapy for Liver Disease

Human Gene Therapy

Volumn 27, Issue 12, 2016, Pages 947-961

  Kattenhorn, Lisa M ^a   Tipper, Christopher H ^a   Stoica, Lorelei ^a   Geraghty, Deborah S ^a   Wright, Teresa L ^a   Clark, K Reed ^a   Wadsworth, Samuel C ^a  

^a ULTRAGENYX PHARMACEUTICAL INC   (United States)

Author keywords

AAV; clinical trials; gene therapy; hemophilia; liver; metabolic

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR;

CANCER RISK; CELLULAR DISTRIBUTION; CODON; GENE TRANSFER; HUMAN; IMMUNE RESPONSE; LIVER CELL; LIVER CELL CARCINOMA; LIVER DISEASE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRECLINICAL STUDY; PROMOTER REGION; REVIEW; SEROTYPE; VIRAL GENE THERAPY; ADMINISTRATION AND DOSAGE; ANIMAL; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; LIVER DISEASES;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LIVER DISEASES;

EID: 85006969361     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.160     Document Type: Review

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