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URMC / Research / Research@URMC / June 2013 / Bird Song Points to Potential New Huntington’s Treatment

Bird Song Points to Potential New Huntington’s Treatment

Mimicking the biology of canaries, researchers have successfully reprogrammed the brain’s native stem cells to create neurons capable of replacing the cells lost in Huntington’s disease.  The experiments – which were done in both mice and squirrel monkeys – could point to a new treatment strategy for the disease.

CanarySome of the key insights for the new findings emerged from a decades-long effort to understand the brains of canaries.  As a graduate student in the early 1990s, URMC neurologist Steve Goldman, M.D., Ph.D. first began to study the birds because of their ability – unique in the animal kingdom – to lay down new networks of neurons into adulthood.  This occurred in the part of the brain where the canaries learned new songs. 

His work – which continues to this day – provided a key insight into the chemical signaling of stem cells found in the brain.  It showed that a protein called brain derived neurotrophic factor (BDNF) was active in the area of the brain where the birds generated new neurons.  This protein is present in humans during early development, however, shortly after birth this signal fades and the brain’s stem cells instead turn their production activity over from creating neurons to pumping out glia – an abundant family of support cells found in the brain. 

Goldman and his colleagues discovered that when the instructions to create BDNF along with another protein called noggin where delivered to the brain using a modified virus, that the neural stem cells would again turn out medium spiny motor neurons (the cells lost in Huntington’s), migrate to the part of the brain ravaged by the disease (the striatum), and integrate with existing neural networks. In some instances, the treatment doubled the life expectancy of the mice – a remarkable achievement. 

You can read more about the study, which appeared today in the journal Cell Stem Cell, here.

Mark Michaud | 6/6/2013

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