Way to Inhibit Neurodegeneration in Adult Form of Fragile X Syndrome Discovered

Expression of a toxic mRNA that leads to Fragile X Tremor Ataxia Syndrome is modified by genetic or pharmacological means, according to recent research from U-M Medical School scientists.

Peter K. Todd led a team of researchers who examined the expression of a toxic messenger RNA (mRNA) seen in the brains of those afflicted with the syndrome.

Ataxia - Causes - Symptoms - Diagnosis - Treatment
Ataxia affects coordination. Gait becomes unstable and the patient loses balance. The cerebellum or its inputs and/or outputs are affected.

Fragile X Tremor Ataxia Syndrome (FXTAS) is usually found in older adults, who often have grandchildren afflicted with Fragile X.

"We found that the expression of this toxic mRNA is dynamic and modifiable. There is a potential for modifying the increased production of the toxic RNA with drugs that inhibit histone acetylation," said Todd.

Using both fruit fly models and human cells, the U-M researchers found that drugs that inhibit histone acetyltransferases modify the brain changes associated with FXTAS and could provide the pathway to a therapeutic target.

"These drugs that we used are too toxic for use in patients but the important finding is that we have a better idea of what's driving this syndrome and proof of principle that those brain changes can be modified," said Todd.

The study was published in the journal Public Library Of Science Genetics.

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Source-ANI

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