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Address Cognitive Impairment in Down Syndrome
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DSRTF Awards New $250,000 Research Grant to Dr. Roger Reeves, Johns Hopkins School of Medicine, for Novel Potential Therapeutic Target to
Address Cognitive Impairment in Down Syndrome
The Down Syndrome Research and Treatment Foundation (DSRTF) has announced the award and funding for a major new DSRTF Research Grant to Roger H. Reeves, Ph.D., Professor, Department of Physiology and McKusick-Nathans Institute for Genetic Medicine at Johns Hopkins School of Medicine, to significantly extend research concerning a novel potential therapeutic target involving neurogenesis (nerve cell development) to ameliorate cognitive impairment in Down syndrome. This new grant represents the latest DSRTF research initiative as part of the targeted expansion of the Foundation’s strategy to stimulate and facilitate the most promising biomedical research that will accelerate development of treatments to significantly improve cognition, including memory, learning and speech, for children and adults with Down syndrome.
This new DSRTF-supported research program is building upon recently published research studies by Dr. Reeves and his colleagues, in which they demonstrated that a Down syndrome mouse model, like humans with Down syndrome, develops a disproportionately smaller cerebellum, a major structural region of the brain (1). Importantly, they further discovered that this resulted from a deficit in a specific molecular signaling pathway, the Sonic or “Shh” growth factor pathway, involving postnatal neurogenesis in the cerebellum and that administration of a prototypical drug compound, specifically acting on the “Shh” pathway, could reverse the deficit and essentially normalize the structural development of the cerebellum. The “Shh” pathway, thus, represents a novel potential therapeutic target in Down syndrome. Previously published research has additionally demonstrated deficits in neurogenesis in the Down syndrome mouse model involving the hippocampus, another major region in the brain crucial to learning and memory, and further, that the “Shh” pathway also plays an important role in hippocampal neurogenesis. DSRTF funding for the new research program will significantly extend and accelerate Dr. Reeves’ research to address two critical questions: 1) Does the prototypical drug compound, targeting the “Shh” pathway, correct the neurogenesis deficit in the Down syndrome hippocampus?; and, 2) Does the action of the prototypical drug compound through this potential therapeutic target produce measurable positive effects on learning and memory?
With the initiation of this new DSRTF Research Grant, DSRTF has added a third novel potential therapeutic target to the DSRTF-supported research portfolio, which includes the two new targets recently identified at Stanford University School of Medicine as part of on-going DSRTF-funded research (2,3). These results represent “unprecedented” and dramatic research progress, and underscore the importance and value of generating critical mass for both new ideas and approaches as well as significant funding for cognition research in Down syndrome. These are key elements in DSRTF’s groundbreaking strategy, which has included DSRTF funding more than $2 million in new research on cognition in Down syndrome over the past three years together with the insight and guidance of the highly distinguished members of the Foundation’s Scientific Advisory Board.
DSRTF recognizes these new and continuing research initiatives and advances would not be possible without the generous financial support of the Foundation’s donors, and is grateful to all those making this important research possible. Additional information and updates on DSRTF and the research supported by the Foundation are available by clicking here.
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