Progress towards gene therapy for the hemophilias.
A variety of vector systems and approaches for transferring the genes for factors VIII and IX are being investigated. This indicates that there is no clearly superior technique that has emerged. However, considerable progress is being made in factor expression in animal models. In addition, as mechanisms of vector biology are elucidated, this facilitates more rational design of vector improvements.
Thompson
A R
AR
University of Washington, Seattle, USA.
D000818:Animals; D017690:Cell Transplantation; D002478:Cells, Cultured / Q000637:transplantation; D005164:Factor IX / Q000235:genetics; D005169:Factor VIII / Q000235:genetics; D005260:Female; D015870:Gene Expression; D015316:Genetic Therapy / Q000639:trends; D005822:Genetic Vectors / Q000145:classification; D006467:Hemophilia A / Q000145:classification / Q000235:genetics / Q000628:therapy*; D006801:Humans; D008297:Male; D051379:Mice; D008822:Mice, Transgenic; D009928:Organ Specificity; D011994:Recombinant Proteins / Q000235:genetics
D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX