alexa The Need for Gene Therapy for the Effective Treatment of Hemophilia
ISSN: 2157-7412

Journal of Genetic Syndromes & Gene Therapy
Open Access

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Short Communication

The Need for Gene Therapy for the Effective Treatment of Hemophilia

Tung Wynn1*, William B Slayton1 and Roland W Herzog2

1Department of Pediatrics, Division of Hematology/Oncology, University of Florida, USA

2Department of Pediatrics, Division of Microbiology and Molecular Genetics, University of Florida, USA

*Corresponding Author:
Tung Wynn, MD
Department of Pediatrics
Division of Hematology/Oncology
University of Florida, USA
Tel: (352)273-9120
Fax: (352)392-8725
E-mail: [email protected]

Received date: August 17, 2012; Accepted date:August 18, 2012; Published date: August 20, 2012

Citation: Wynn T, Slayton WB, Herzog RW (2012) The Need for Gene Therapy for the Effective Treatment of Hemophilia. J Genet Syndr Gene Ther S1:016. doi:10.4172/2157-7412.S1-016

Copyright: © 2012 Wynn T, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.



Gene therapy is promising to patients with hemophilia, with or without inhibitors. It offers the promise of life without the risk of bleeding after a simple injury and chronic arthropathy. It provides the promise of life without IV infusions for factor replacement. It makes the promise that no person with hemophilia will have to live a tragic life. Restoring a patient’s ability to make a protein that was congenitally deficient and all but curing the disorder is the goal of everyone treating any heritable disease and is the goal of gene therapy for hemophilia patients. Ultimately, gene therapy promises life without hemophilia. The goal and promise of gene therapy is well worth striving for.


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