Viral vector-mediated gene therapy for hemophilia B.
Over the past five years, significant advances have been made in the development of novel viral vector systems for the treatment of hemophilia B by somatic gene therapy. At present, both a sustained but partial or a complete but transient correction of the hemophilia B phenotype have been observed in a clinically relevant animal model. Present efforts are being directed toward the development of safe, effective and persistent methods of virally-mediated gene transfer to achieve the complete restoration of normal hemostasis in individuals with hemophilia B.
Eisensmith
R C
RC
Institute for Gene Therapy and Molecular Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA. eisenr01@doc.mssm.edu
Woo
S L
SL
D000256:Adenoviridae / Q000235:genetics*; D005164:Factor IX / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans; D012190:Retroviridae / Q000235:genetics*
D005164:Factor IX