The has announced a gift of $3.4 million from the in Brazil for a research collaboration with aimed at advancing understanding of and therapies for Down syndrome.
To be led by Professor Li-Huei Tsai, director of the at MIT, and Professor Alberto Costa, director of the at CWRU's School of Medicine, the effort will focus on the development of personalized human stem cell models of Down syndrome called iPSCs (induced pluripotent stem cells) that can be induced to develop into different types of mature cells such as neurons. Researchers can then study the induced human neuronal models to better understand the biology of the genetic condition. From there, the team will use the human neuronal model to test the benefits of new therapeutic treatments, which could provide a preclinical drug screening approach that is personalized to the condition in specific individuals.
As they age, people with Down syndrome are at greater risk than the general public to develop a type of dementia similar to Alzheimer's disease. With that in mind, the grants also will support efforts by researchers to determine whether the same pathological processes that are at work in Alzheimer's occur in Down syndrome. In a fourth project, the teams will use a 3-D imaging technique invented by Kwanghun Chung, an assistant professor of chemical engineering at MIT, to study the circuits, anatomy, and pathologies of the mouse model of Down syndrome, as well as human tissue models of the condition.
"There is tremendous opportunity to benefit from our combined strengths — MIT's ongoing research to understand the aging brain, and Case Western Reserve's expertise in Down syndrome," said Tsai, who is also the Picower Professor of Neuroscience in MIT's . "The Alana Foundation's support will allow us to work together to accelerate research projects with the potential to develop treatments that will improve the quality of life for individuals with Down syndrome."