Gene therapy in hemophilia: clinical trials update. Thromb Haemost 2001 Jul;86(1):172-7
Date
08/07/2001Pubmed ID
11487005Scopus ID
2-s2.0-0034923893 (requires institutional sign-in at Scopus site) 27 CitationsAbstract
Disorders caused by inborn genetic errors have been a primary target for treatment by gene transfer. Hemophilia A and B have been considered especially important targets because the genes for factor VIII and IX are well characterized, levels of factor VIII and IX do not require complex regulation, small increases in factor level would have significant clinical benefits, good clinical and laboratory tests of efficacy exist, and excellent animal models of hemophilia are available. Four clinical trials of gene transfer in hemophilia, two in hemophilia A and two in hemophilia B, are currently underway or have been completed and two other trials have been approved. The collective interim results from these trials indicate that the current approaches and doses are safe and that low levels of expression are detected. These studies support the continued development of gene transfer as a potential treatment option for hemophilia.
Author List
White GC 2ndAuthor
Gilbert C. White MD Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
Clinical Trials as TopicGene Transfer Techniques
Genetic Therapy
Genetic Vectors
Hemophilia A
Hemophilia B
Humans
