Current and future prospects for hemophilia gene therapy

Expert Review of Hematology

Volumn 9, Issue 7, 2016, Pages 649-659

  Ward, Peter ^a   Walsh, Christopher E ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Aav; Clinical trial; Gene therapy; Genotoxicity; Hemophilia

Indexed keywords

ADENOVIRUS VECTOR; GENOMIC DNA; NEUTRALIZING ANTIBODY; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9;

ADENO ASSOCIATED VIRUS; CARCINOGENICITY; CLINICAL TRIAL (TOPIC); DISEASE PREDISPOSITION; GENE TARGETING; GENOTOXICITY; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IMMUNE RESPONSE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; REVIEW; SEROTYPE; TARGETED GENE REPAIR; TRANSLATIONAL RESEARCH; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM; VIRUS RECOMBINANT; ADVERSE EFFECTS; ANIMAL; CLASSIFICATION; DEPENDOPARVOVIRUS; GENE EDITING; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; PRECLINICAL STUDY; PROCEDURES;

ANIMALS; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; DRUG EVALUATION, PRECLINICAL; FACTOR IX; FACTOR VIII; GENE EDITING; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA A; HEMOPHILIA B; HUMANS; TARGETED GENE REPAIR;

EID: 84977137707     PISSN: 17474086     EISSN: 17474094     Source Type: Journal    
DOI: 10.1080/17474086.2016.1182859     Document Type: Review

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