Newly Published Results Describe Restored Cerebellar Function in Down Syndrome Mouse Model

Research Down Syndrome has been communicating progress on research funded by RDS and other organizations being conducted at Johns Hopkins University in the laboratory headed by Dr. Roger Reeves. This research has just been published in the scientific journal, Science Translational Medicine.

The study describes how a single treatment of newborn Ts65Dn mice with the Sonic hedgehog pathway agonist(or response stimulator) SAG 1.1 results in normal cerebellar morphology in adults. (The Ts65Dn Down syndrome mouse model has triplicate copies of many of the genes associated with Human chromosome 21, and the Sonic Hedgehog Pathway is an important contributor to brain cell development in the cerebellum in humans.) Also, mice treated at birth with SAG 1.1 showed behavioral improvements and normalized performance in a water maze task for learning and memory.
The positive results from this scientific study suggest a possible direction for therapeutic intervention to improve cognitive function for those with Down syndrome. As with all such animal model studies, however, the researchers caution that SAG 1.1 has yet to be proven effective in humans and that "Before a clinical application is contemplated in people with DS, it will be necessary to better understand the SAG 1.1 role in hippocampal function and the sensitivity to possible side effects on different genetic backgrounds while refining both the dosage and the route of drug administration". has authored an excellent summary of this paper. 

RDS recognizes this extraordinary achievement by Dr. Reeves and his team. The study adds yet more tangible evidence to the rapid and exciting progress of Down syndrome cognitive research. Your support is more important than ever in helping sustain the momentum of this remarkable research initiative.