AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B - Manno, Catherine S.; Chew, Amy J.; Hutchison, Sylvia; Larson, Peter J.; Herzog, Roland W.; Arruda, Valder R.; Tai, Shing Jen; Ragni, Margaret V.; Thompson, Arthur; Ozelo, Margareth; Couto, Linda B.; Leonard, Debra G.B.; et al. (Blood, 2003)
Keywords: Adult; Aged; Amino Acid Substitution; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Applied cell therapy and gene therapy; Biological and medical sciences; Biopsy; Combined Modality Therapy; Dependovirus - genetics; Factor IX - analysis; Factor IX - genetics; Factor IX - therapeutic use; Feasibility Studies; Genetic Therapy; Genetic Vectors - administration & dosage; Genetic Vectors - therapeutic use; Hematologic and hematopoietic diseases; Hemophilia B - complications; Hemophilia B - genetics; Hemophilia B - therapy; Hepatitis, Viral, Human - complications; HIV Infections - complications; Humans; Injections, Intramuscular; Male; Medical sciences; Muscle, Skeletal - metabolism; Muscle, Skeletal - pathology; Mutation, Missense; Platelet diseases and coagulopathies; Recombinant Fusion Proteins - analysis; Safety; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Human; Recombinant virus; Hemophilia B; Hemopathy; Dependovirus; Transgene; Genetic disease; Genetic transfer; Virus; Factor IX; Treatment; Parvoviridae; Phase I trial; Gene therapy; Vector; Coagulopathy; Parvovirinae