434e6f50eb5a8dabe00f189eb660722f_f92A Message From Dr. Steve Gray, PhD
Gene Therapy Center, UNC Chapel Hill

Before Hannah’s Hope Fund existed, I was doing research for Rett Syndrome funded by IRSF. Rett Syndrome is fatal in boys, who die in the first year if they come to term. 1 in 10,000 girls have this (I can explain the genetics, but just take my word for it). It is considered a form of autism; after developing normally for the first 1-2 years, girls will lose the gains they’ve made and suffer from often severe mental retardation and other symptoms such as breathing irregularities that can cause premature death.

This is a very difficult disease to treat, 1) because it affects the entire brain and 2) the missing gene (MeCP2) affects literally hundreds of other genes causing a large range of problems. If a gene therapy approach can be worked out similar to the one we’re doing for GAN, it offers the potential to solve the problem at the source. IRSF has also made considerable headway researching various drugs that might alleviate specific symptoms. Part of my research with IRSF led me to solve a critical problem in our development of a GAN treatment.

While I celebrated HHF’s victory, I was also sad that the IRSF couldn’t get the money too. After thinking HHF lost Tuesday night, I think you can understand how they are feeling right now. If you can keep your enthusiasm going another month, please throw your support behind them.
http://www.refresheverything.com/rettresearchtoreality
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Thank you,
Steve