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Advanced therapies for the treatment of hemophilia: future perspectives

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Liras, Antonio and Segovia, Cristina and Gaban, Aline S. (2012) Advanced therapies for the treatment of hemophilia: future perspectives. Orphanet journal of rare diseases, 7 . pp. 1-9. ISSN 1750-1172, 1750-1172(ESSN)

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Official URL: http://www.ojrd.com/content/7/1/97


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Abstract

Monogenic diseases are ideal candidates for treatment by the emerging advanced therapies, which are capable of correcting alterations in protein expression that result from genetic mutation. In hemophilia A and B such alterations affect the activity of coagulation factors VIII and IX, respectively, and are responsible for the development of the disease. Advanced therapies may involve the replacement of a deficient gene by a healthy gene so that it generates a certain functional,structural or transport protein (gene therapy); the incorporation of a full array of healthy genes and proteins through perfusion or transplantation of healthy cells (cell therapy); or tissue transplantation and formation of healthy organs (tissue engineering). For their part, induced pluripotent stem cells have recently been shown to also play a significant role in the fields of cell therapy and tissue engineering.Hemophilia is optimally suited for advanced therapies owing to the fact that, as a monogenic condition, it does not require very high expression levels of a coagulation factor to reach moderate disease status. As a result, significant progress has been possible with respect to these kinds of strategies, especially in the fields of gene therapy (by
using viral and non-viral vectors) and cell therapy (by means of several types of target cells). Thus, although still considered a rare disorder, hemophilia is now recognized as a condition amenable to gene therapy, which can be administered in the form of lentiviral and adeno-associated vectors applied to adult stem cells, autologous fibroblasts, platelets and hematopoietic stem cells; by means of non-viral vectors; or through the repair of mutations by chimeric oligonucleotides. In hemophilia, cell therapy approaches have been based mainly on transplantation of healthy cells (adult stem cells or induced pluripotent cell-derived progenitor cells)in order to restore alterations in coagulation factor expression.


Item Type:Article
Uncontrolled Keywords:Advanced therapies, Gene therapy, Cell therapy, Hemophilia A, Hemophilia B
Subjects:Medical sciences > Medicine > Pharmacology
Medical sciences > Medicine > Medical genetics
Medical sciences > Medicine > Hematology
ID Code:30778
Deposited On:12 Jun 2015 11:08
Last Modified:06 Jul 2015 12:02

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