We’ve defined a clear path to drugs and interventions.
Research studies currently funded — either in whole or part — by LuMind RDS, target each of our four strategic scientific categories that span four independent but interlinked life-stages, beginning during prenatal development and extending into old age.
Targeting these important developmental periods in the lives of people with Down syndrome may be a more successful strategy for improving overall quality of life. As an individual grows and matures, there are unique processes that underlie cognitive development and function.
Preventing Alzheimer’s onset
- LuMind RDS intends to support industry-led clinical trials of therapeutic approaches by: supporting the development of adaptive assessment scales, which account for the variation in symptoms; establishment of a clinical trial network, and a developing a registry for rapid enrollment in trials.
- To gain a better understanding of cognition in Down syndrome, LuMind RDS will support research on early intervention; validation of specific cognitive targets, the unique sleep patterns and techniques to improve sleep among those with Down syndrome; and finding anti-inflammatory treatments that target specific genes associated with interferon.
Developing gene therapy
- Gene therapy is an exciting avenue for possible treatment. In females, the second X chromosome is silenced. Research into some specific genes, Dyrk1A and USP16, may show that the same could be achieved with the third 21st chromosome. Research will be funded on restoring the right level of specific gene(s).
- Discover novel targets (stem cells, sequencing/phenotyping)
Improve mouse models to better test novel targets
Blazing a trail to
conduct large, multi-site
trials for Down syndrome.
Stem cell research using
skin cells of individuals
with and without DS
MIT - Picower Institute