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Brain derived neurotrophic factor alleviates disease symptoms in a mouse model of Rett Syndrome

In fact the mice were far less lethargic, had slightly larger brains and longer lifespans.

"This is the first instance that the Rett disease progression has been altered by changing expression of another gene. Once we understand the molecular mechanism of BDNF's effect on Rett mice it may be possible in the future to design rational therapies that could alter the disease progression in patients," said Rudolf Jaenisch.

"The next step is to understand how much BDNF overexpression is needed and in which specific parts of the brain," states Qiang Chang.

"Despite remarkable progress in the MECP2/RTT field no obvious "druggable targets" have emerged as candidates for drug development. It's encouraging to see that in these experiments BDNF was able to ameliorate the disease state in mice. Should BDNF turn out to be a druggable target for RTT, RSRF stands poised to help accelerate the transition from lab to clinic," said Monica Coenraads, Co-founder and Director of Research for RSRF.


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Contact: Monica Coenraads
monica@rsrf.org
203-445-9233
Rett Syndrome Research Foundation
1-Feb-2006


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