The Wolfe Lab investigates vector-mediated gene transfer and stem cell transplantation in pediatric neurodevelopmental genetic diseases. This line of investigation involves human and animal induced pluripotent stem (iPS) cell-derived neural stem cells.
One of the main areas of current research in the lab focuses on stem cell-based approaches aimed at restoring vision. In this multidisciplinary study co-led by the Wolfe Lab, the investigators aim to build the foundation for cell-based treatments to restore vision to people who have lost key vision cells.
While existing gene therapies have had success in treating blindness, they cannot save areas of the retina where cells have already died. The Wolfe Lab’s research project plans to reprogram cells taken from adult animals into stem cells, culture the stem cells in the lab into retinal photoreceptor cells, and then transfer the cells into the retinas of dogs with vision loss. The researchers will then determine how well the implanted cells function.
The Wolfe Lab has made significant contributions to testing and developing gene therapy approaches, particularly for intellectual developmental disorders. The lab investigates gene therapy approaches to treating lysosomal storage disorders, which require global brain correction.