Research Awards
Rudolf Jaenisch, M.D.
Whitehead Institute for Biomedical Research
"Mecp2 Deficiency in Mice: Can the Course of RTT-like Disease Development be Modified?"
2-Year Award: $100,000
Lay Progress Report (August, 2003)
We are using the MeCP2 mutant mice as a model system to study the molecular mechanism of human Rett Syndrome and to design therapeutic approaches. We have focused on identifying target genes of MeCP2, and assessing whether any of them is of therapeutic potential. We have found BDNF (Brain Derived Neurotrophic Factor) to be such a gene. We have reason to believe that BDNF might play a role in the progression of RTT disease. We are doing more research to test whether manipulating BDNF expression in MeCP2 mutant mice can slow down disease progression. We are also using various transgenic approaches to reactivate Mecp2 in mutant mice at different stages of disease progression.
Rudolf Jaenisch, M.D.Whitehead Institute for Biomedical Research
"Mecp2 Deficiency in Mice: Can the Course of RTT-like Disease Development be Modified?"
2-Year Award: $100,000
Lay Progress Report (August, 2003)
We are using the MeCP2 mutant mice as a model system to study the molecular mechanism of human Rett Syndrome and to design therapeutic approaches. We have focused on identifying target genes of MeCP2, and assessing whether any of them is of therapeutic potential. We have found BDNF (Brain Derived Neurotrophic Factor) to be such a gene. We have reason to believe that BDNF might play a role in the progression of RTT disease. We are doing more research to test whether manipulating BDNF expression in MeCP2 mutant mice can slow down disease progression. We are also using various transgenic approaches to reactivate Mecp2 in mutant mice at different stages of disease progression.