New protein therapy approach to be tested in fragile X mouse model
$100K FRAXA grant will support studies of brain activity at UC Riverside
Written by |
The FRAXA Research Foundation has awarded a $100,000 grant to fund a research project that will test Bowen’s FX Therapeutics’ experimental protein-replacement therapies for fragile X syndrome in mice.
The newly funded research will be conducted in the laboratory of Devin Binder, MD, PhD, a professor at the University of California (UC), Riverside. Binder’s lab has studied fragile X and brain activity in mouse models for years.
“I see this project as an example of how foundation funding can bring together academic neuroscience and biotechnology,” Binder said in a news story from UC Riverside. “Bowen’s FX Therapeutics provides access to a promising therapeutic technology, while my laboratory brings expertise in measuring brain activity in living animals. As academics, we otherwise wouldn’t necessarily have access to these kinds of therapeutics.”
Protein approach aims to restore FMRP in the brain
Fragile X syndrome is a genetic disorder caused by mutations in the FMR1 gene, which provides instructions for making FMRP, a protein important for normal brain development and function. Reduced or absent working FMRP is thought to contribute to the neurological symptoms of fragile X.
Bowen’s FX is developing engineered versions of FMRP designed to be given systemically and cross the blood-brain barrier, which limits the passage of many substances from the bloodstream into the brain. The goal is to deliver working FMRP to the brain as a potential treatment for people with fragile X.
“What our collaborators are doing is possible now in a way that I never thought was possible when I first entered this field,” Binder said. “They can engineer a protein and get it into the brain and cells. A few years ago, that would have seemed like science fiction.”
Binder’s lab has previously identified abnormalities in electroencephalogram (EEG) recordings — measures of electrical activity in the brain — in a well-established fragile X mouse model. The new study will test Bowen’s FX therapy candidates to see whether they can help normalize that brain activity.
“We’re looking at the whole animal as it’s actually behaving and processing stimuli,” Binder said. “Our goal is to obtain very high-quality brain recordings from the mice.”
The study will not directly determine whether treated mice improve in learning or memory. But if the treatment corrects abnormalities in brain activity, Binder said some related cognitive and behavioral problems could also improve.
“If the Fragile X mice after treatment begin to resemble the normal mice, if their physiology is essentially normalized, that would be a very significant result,” he said.
Gwenn
This is very interesting and something I though would be effective for a long time now. My question is what vector would be needed for the engineered FMRP to be able to cross the blood brain barrier? How is this possible and is it safe?