Human stromal stem cell therapy using gene-modified cells

Abstract

There is an increasing demand to develop novel approaches for the treatment of a large number of chronic degenerative diseases affecting primarily the aging population and where there is currently no effective therapy, e.g., Parkinson’s disease, liver failure, diabetes, osteoarthritis, and osteoporosis. An emerging therapeutic approach for the management of these conditions is cell therapy or cellular therapeutics where organ functions are restored through transplanting healthy, functional cells. Stem cells, because of their nature, are currently considered the most suitable cells for cell therapy. As mentioned in other chapters in this book, gene therapy aims at modifying specific characteristics of target cells with possible therapeutic effects. Thus, combining gene therapy with stem cell therapy provides an additional useful dimension to the use of stem cells for treatment. Stromal (mesenchymal) stem cells are being introduced into clinical trials due to their ease of isolation and efficacy in treating a number of disease conditions in animal preclinical disease models. Also, there are an increasing number of studies that have tested the genetically modified mesenchymal stem cells (MSC) functionality in vivo. The aim of this chapter is to review the potential use of gene-modified stem cells, in particular gene-modified MSC, in therapy and the challenges facing their use in clinical practice.