Down Syndrome

Down syndrome is the most prevalent genetic disorder, occurring in one of every 700 live births. Until recently a therapeutic for individuals with Down syndrome seemed improbable, due to the large number of genes (extra chromosome 21) that could be responsible for the developmental disorder. Studies suggest that a 33-gene region of the chromosome appears critical for the cognitive deficit and that potentially one gene, Dyrk1a, may play a partial in the developmental cognitive impairment.

Our neurogenic therapeutic candidate for depression and post-traumatic stress disorder, NNI-351, also inhibits a protein, Dyrk1a protein, that is overproduced in Down syndrome individuals. The inhibition of the protein appears to play some role in how NNI-351 increases the production of new neurons in the hippocampus (area of attention, learning and memory), thus reducing the overall hyperactivity and increasing the learning and memory ability in Down syndrome transgenic mice. Of great importance, is that these beneficial effects of NNI-351 are disease-modifying and do not just mask the symptoms in this well established mouse model of Down syndrome. As stated elsewhere, a patent for NNI-351 has now been issued to Neuronascent by the United States patent office.

For more information on Down syndrome, please visit the NICHD website at http://www.nichd.nih.gov/health/topics/down/Pages/default.aspx.

Increase in Neurons in Mice from NNI-351