Oral Presentation 2014 Cutaneous Biology Meeting

Exploring the Therapeutic Potential of Induced Pluripotent Stem (iPS) Cells for Inherited Skin Diseases (#3)

Dennis Roop 1
  1. Charles C. Gates Center for Regenerative Medicine and Stem Cell Biology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, United States

Currently, no effective therapeutic treatments are available for the majority of genetic skin diseases. The ability to produce induced pluripotent stem cells (iPSCs) from skin biopsies of patients with inherited skin diseases provides a renewable source of cells with embryonic stem cell (ESC)-like properties, which may potentially be used for corrective gene therapy and tissue engineering. Although many obstacles remain to be solved before these cells can be applied clinically, the development of efficient protocols for the differentiation of iPSCs into particular cell types and tissues is a prerequisite for their future application. My laboratory has recently been successful in developing a method for the efficient differentiation of human iPSCs into a keratinocyte lineage through subsequent applications of retinoic acid and bone-morphogenetic protein-4. The development of a method for the differentiation of iPSCs into a keratinocyte lineage will now enable us to determine whether genetically corrected iPSCs can be used to generate a permanent corrective therapy for inherited skin diseases. We are currently generating iPSCs from patients with inherited skin diseases using methods which do not require viral vectors, and determining whether Zinc Finger Nucleases and Transcription Activator-like Effector Nucleases (TALENS) can be used to correct the genetic defect in these patient-specific iPSCs.