AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Blood

Volumn 101, Issue 8, 2003, Pages 2963-2972

  Manno, Catherine S ^a   Chew, Amy J ^b   Hutchison, Sylvia ^b   Larson, Peter J ^b   Herzog, Roland W ^b   Arruda, Valder R ^b   Tai, Shing Jen ^b   Ragni, Margaret V ^b   Thompson, Arthur ^b   Ozelo, Margareth ^b   Couto, Linda B ^b   Leonard, Debra G B ^b   Johnson, Frederick A ^b   McClelland, Alan ^b   Scallan, Ciaran ^b   Skarsgard, Erik ^b   Flake, Alan W ^b   Kay, Mark A ^b   High, Katherine A ^b   Glader, Bertil ^b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; VIRUS VECTOR; HYBRID PROTEIN;

ADENO ASSOCIATED VIRUS; ARTICLE; CIRCULATION; CLINICAL ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; FEMALE; GENE EXPRESSION; GENE TRANSFER; GENOME; HEMOPHILIA B; HUMAN; IMMUNOHISTOCHEMISTRY; MALE; MISSENSE MUTATION; MUSCLE BIOPSY; PRIORITY JOURNAL; SIGNAL TRANSDUCTION; SKELETAL MUSCLE; TRANSGENE; ADULT; AGED; AMINO ACID SUBSTITUTION; BIOPSY; FEASIBILITY STUDY; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INTRAMUSCULAR DRUG ADMINISTRATION; METABOLISM; MULTIMODALITY CANCER THERAPY; PATHOLOGY; SAFETY; VIRUS HEPATITIS;

ADULT; AGED; AMINO ACID SUBSTITUTION; BIOPSY; COMBINED MODALITY THERAPY; DEPENDOVIRUS; FACTOR IX; FEASIBILITY STUDIES; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HEPATITIS, VIRAL, HUMAN; HIV INFECTIONS; HUMANS; INJECTIONS, INTRAMUSCULAR; MALE; MUSCLE, SKELETAL; MUTATION, MISSENSE; RECOMBINANT FUSION PROTEINS; SAFETY;

EID: 0013442744     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2002-10-3296     Document Type: Article

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