Davis said the researchers are exploring the use of an experimental treatment involving gene-corrected iPS derived cells generated using zinc finger nuclease (ZFN) technology developed by Sangamo BioSciences, Inc. to address these two conditions.
"We're going to start with the skin cells of a patient with a lung disease, then take those cells and convert them into iPS cells and correct the genetic defect. Then finally, the disease free iPS cells will be used to generate lung cells that can be used for cell-based therapies," he said.
One of the benefits of this technique, according to Wetsel, is that because researchers are using the patient's own cells, it is far less likely that the patient's immune system will reject the gene-corrected cells. Wetsel is the William S. Kilroy Sr. Chair in Pulmonary Disease at the IMM and he is also the director of the Hans J. Mueller-Eberhard & Irma Gigli Research Center for Immunology and Autoimmune Diseases at the IMM.
The research funded by the second NIH award will support research in animal models. If these studies are successful, they may lay the foundation for the next generation of preclinical studies designed to explore the effectiveness of these approaches for eventually treating pulmonary diseases in patients.
C. Thomas Caskey, M.D., director and CEO of the IMM, said, "These awards recognize significant developments in two areas. The first award addresses issues critical to blood stem cell derivation from pluripotent stem cells with the potential to greatly benefit transplantation for various diseases of the blood forming system. The second award involves the conversion of skin cells to pluripotent stem cells from an individual with lung disease, and in common with the first award, enables personalized therapeutic initiatives. Both programs were enabled by Houston philanthropy p
|Contact: Robert Cahill|
University of Texas Health Science Center at Houston