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Mesenchymal stem cell treatment for hemophilia: a review of current knowledge
Author(s) -
Sokal E. M.,
Lombard C.,
Mazza G.
Publication year - 2015
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/jth.12933
Subject(s) - mesenchymal stem cell , genetic enhancement , medicine , stem cell , clotting factor , cell therapy , immune system , factor ix , clinical trial , cell , immunology , gene , biology , pathology , microbiology and biotechnology , genetics
Summary Hemophilia remains a non‐curative disease, and patients are constrained to undergo repeated injections of clotting factors. In contrast, the sustained production of endogenous factors VIII ( FVIII ) or IX ( FIX ) by the patient's own cells could represent a curative treatment. Gene therapy has thus provided new hope for these patients. However, the issues surrounding the durability of expression and immune responses against gene transfer vectors remain. Cell therapy, involving stem cells expanded in vitro , can provide de novo protein synthesis and, if implanted successfully, could induce a steady‐state production of low quantities of factors, which may keep the patient above the level required to prevent spontaneous bleeding. Liver‐derived stem cells are already being assessed in clinical trials for inborn errors of metabolism and, in view of their capacity to produce FVIII and FIX in cell culture, they are now also being considered for clinical application in hemophilia patients.