Management of hemophilias--gene therapy to come?
BACKGROUND
Hemophilias are relatively common coagulopathies that require very careful management to prevent bleeding and related complications.


OBJECTIVE
To review the current management of hemophilias, with an overview of gene therapy.


CONCLUSIONS
Although with the availability of highly purified and recombinant factor concentrates the replacement therapy of hemophilias has become safer and more effective, a therapeutic method providing continuous supply of clotting factors is desirable. Theoretically, this is possible, by transplanting organs, than can express antihemophilic proteins, but in most cases a less invasive method is preferable. Therefore the gene therapy would be revolutionary in treating hemophiliacs.


CONCLUSIONS
The treatment of choice for hemophilias is the infusion of highly purified or recombinant factor products. Although still in experimental level, gene therapy for hemophilias is promising and may become the treatment of choice in these disorders.
Vajó
Z
Z
Department of Medicine, Maricopa Medical Center, Phoenix, Arizona, USA.
Sréter
L
L
D001803:Blood Transfusion; D002460:Cell Line; D005164:Factor IX / Q000627:therapeutic use; D005169:Factor VIII / Q000627:therapeutic use; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006467:Hemophilia A / Q000235:genetics / Q000628:therapy*; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use; D012190:Retroviridae
D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX