Igor Slukvin, MD, PhD, University of Wisconsin School of Medicine

"Induced Pluripotent Stem (iPS) Cell-Based Therapy for SLE"

2009 Adult Stem Cell Grant Award

The major goal of this proposal is to significantly advance the stem cell-based therapies for drug-resistant lupus through development of novel technologies for generation of HSCs and immunotherapeutic cells from induced pluripotent cells (iPSCs). The iPSCs are stem cells artificially derived from somatic fibroblasts through insertion of genes critical for the maintenance of pluripotency. These cells can be generated from SLE patients and differentiated into blood cells. Because iPSCs and sustainably expanded and genetically altered in a highly controlled way that is not yet possible with more committed HSCs, iPSCs could provide important advantages over somatic HSCs as target cells for stem cell based therapy of SLE. In this application we propose to characterize in vivo repopulating potential of primitive hematopoietic cells derived from iPSCs, develop a system for differentiation of iPSCs into dendritic cells and T cells, and demonstrate the feasibility of iPSC-based gene transfer into hematopoietic cells. The proposed studies could ultimately lead to novel adult stem cell-based and gene therapies for SLE.