Disorder-Related Research

At the Waisman Center, researchers utilize stem cells to advance knowledge about brain development and to identify new treatments and therapies for many complex conditions.

Neurodegenerative diseases (such as Parkinson’s disease, Amyotrophic Lateral Sclerosis, and Alexander disease), are characterized by damage to the central nervous system and a decline in motor function. This is an area of research for which scientists hope iPS cell therapies hold the most potential. Previous research lead scientists to believe that once nerve cells were destroyed, there was no hope for recovery. Scientists now believe the brain is much more “plastic” than previously thought and there is potential to repair or replace damaged neural cells. Studies are also underway to use iPS cells to model retinal degenerative diseases such as macular degeneration and retinitis pigmentosa and to develop treatments for these blinding disorders.

Developmental disabilities, such as autism, Down syndrome, fragile X syndrome, and Rett syndrome are also being studied for the potential therapeutic benefits of iPS cells. Disorder-specific stem cells are used to gain insight into the specific disruptions in brain development that may occur in these disabilities.