Obstacles and future of gene therapy for hemophilia

Expert Opinion on Orphan Drugs

Volumn 3, Issue 9, 2015, Pages 997-1010

  Arruda, Valder R ^a,b   Samelson Jones, Ben J ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Adeno associated viral; Gene therapy; Genetic disease; Hemophilia; Recombinant vectors

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; NEUTRALIZING ANTIBODY; RETROVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; CELLULAR IMMUNITY; CHILD; DRUG MANUFACTURE; ECTOPIC DIRECTED GENE THERAPY; EX VIVO STUDY; FUTUROLOGY; GENOTOXICITY; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IN VIVO STUDY; MULTICENTER STUDY (TOPIC); NONHUMAN; PATIENT SAFETY; PEDIATRICS; PRIORITY JOURNAL; REVIEW; VIRAL GENE THERAPY; VIRUS IMMUNITY; VIRUS INHIBITION; VIRUS TRANSMISSION;

EID: 84946607094     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1069179     Document Type: Review

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