alexa Treatment of Hemophilia A in Utero and Postnatally usin
ISSN: 2157-7412

Journal of Genetic Syndromes & Gene Therapy
Open Access

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Review Article

Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery

Christopher D. Porada* and Graça Almeida-Porada

Wake Forest Institute for Regenerative Medicine, Winston-Salem, NC, USA

*Corresponding Author:
Christopher D. Porada
Regenerative Medicine
Wake Forest Institute for Regenerative Medicine
Richard H. Dean Biomedical Building
391 Technology Way, Winston-Salem
NC 27157-1083, USA
Tel: 336-713-1631
E-mail: [email protected]

Received date: April 06, 2012; Accepted date:May 23, 2012; Published date: May 25, 2012

Citation: Porada CD, Almeida-Porada G (2012) Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery. J Genet Syndr Gene Ther S1:011. doi:10.4172/2157-7412.S1-011

Copyright: © 2012 Porada CD, 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.

 

Abstract

Hemophilia A represents the most common inheritable deficiency of the coagulation proteins. Current stateof- the-art treatment consists of frequent prophylactic infusions of plasma-derived or recombinant FVIII protein to maintain hemostasis, and has greatly increased life expectancy and quality of life for many hemophilia A patients. This treatment approach is, however, far from ideal, due to the need for lifelong intravenous infusions, the high treatment cost, and the fact that it is unavailable to a large percentage of the world’s hemophiliacs. There is thus a need for novel treatments that can promise long-term or permanent correction. In contrast to existing protein based therapeutics, gene therapy offers to provide a permanent cure following few, or even a single, treatment. In the present paper, we review ongoing work towards this end, focusing on studies we have performed in a large animal model. Some of the key topics covered in this review include the unique opportunities sheep offer as a model system, the re-establishment and clinical and molecular characterization of a line of sheep with severe hemophilia. A, the advantages and feasibility of treating a disease like hemophilia A in utero, and the use of Mesenchymal Stem Cells (MSC) as cellular delivery vehicles for the FVIII gene. The review finishes with a brief discussion of our recent success correcting ovine hemophilia A with a postnatal transplant with gene-modified MSC, and the limitations of this approach that remain to be overcome.

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