Hemophilia gene therapy: update.
Gene transfer is an exciting and potentially important treatment approach for hemophilia A and B. Four phase I clinical trials of the safety of gene transfer in hemophilia A or B have been completed and two more trials are currently underway. The results of these trials indicate that gene transfer in hemophilia with the vectors and doses used is safe and well tolerated. Efforts continue to understand the basic biology and improve the efficiency of gene transfer.
Monahan
Paul E
PE
Department of Pediatrics, University of North Carolina at Chapel Hill, 418 MacNider Building, CB#7220 UNC-CH, School of Medicine, Chapel Hill, North Carolina 27599-7220, USA. Paul_Monahan@med.unc.edu
White
Gilbert C
GC
D000818:Animals; D002986:Clinical Trials as Topic; D005164:Factor IX / Q000008:administration & dosage / Q000235:genetics; D005169:Factor VIII / Q000008:administration & dosage / Q000235:genetics; D015316:Genetic Therapy; D005822:Genetic Vectors; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy; D006801:Humans
D005169:Factor VIII; D005164:Factor IX