Induced Pluripotent Stem Cells for the Treatment of Hemophilia ABilgimol C Joseph and Manjunath S Rao*
Micro Therapeutic Research Labs Pvt Ltd, Chennai, Tamilnadu, India
- *Corresponding Author:
- Manjunath S Rao
No: 6, 'Rajam Bhavanam, Kamarajar Salai
East Tambaram, Selaiyur, Chennai - 600 059, India
Tel: +91 44 2239 0070 / 71 / 72
Fax: +91 44 2239 0068
E-mail: [email protected]
Received date: November 01, 2014; Accepted date: November 03, 2014; Published date: November 04, 2014
Citation: Joseph BC, Rao MS (2014) Induced Pluripotent Stem Cells for the Treatment of Hemophilia A. J Cell Sci Ther 5:e119. doi: 10.4172/2157-7013.1000e119
Copyright: © 2014 Joseph BC, 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.
Factor VIII, one of the most complex proteins known, plays a major role in blood coagulation pathway. Defects in factor VIII protein result in hemophilia A, a severe bleeding disorder. Plasma derived factor VIII or recombinant factor VIII has been used extensively for treating hemophilia A patients. Number of attempts at gene therapy for hemophilia A has failed for various unknown/not much studied reasons including immune rejection. Here, the progress that has been made in establishing iPSC-based disease models and the potentials of iPSC technology for personalized medicine and cell therapy for hemophilia A are reviewed. The challenges of iPSC technology are also briefly discussed.