Can I read Gene Therapy for Hemophilia on EtoBox?

Gene Therapy for Hemophilia by Amit C. Nathwani; Andrew M. Davidoff; Edward G.D. Tuddenham is a Medicine article available to read on EtoBox.

What is Gene Therapy for Hemophilia about?

The best currently available treatments for hemophilia A and B (factor VIII or factor IX deficiency, respectively) require frequent intravenous infusion of highly expensive proteins that have short half-lives. Factor levels follow a saw-tooth pattern that is seldom in the normal range and falls so low that breakthrough bleeding occurs. Most hemophiliacs worldwide do not have access to even this level of care. In stark contrast, gene therapy holds out the hope of a cure by inducing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene to replace the hemophilic patient's own defective gene.

Who reads Gene Therapy for Hemophilia?

Author

Amit C. Nathwani; Andrew M. Davidoff; Edward G.D. Tuddenham

Publisher

Elsevier BV

Published

2017

Language

EN

Field

Medicine (Health Sciences)