Postpone Huntington’s disease onset, Discovery of potential biomarker

A new discovery of a potential biomarker for Huntington's disease (HD) which results in the more effective way of evaluating the capability of treatments for this neurological disease. The discovery may provide insight into treatments that could delay the death of neurons in people who carry the Huntington's disease gene mutation, but who do not yet show symptoms of the disease.

Huntington’s Disease is a fatal, inherited neurological disease that usually manifests between 30 and 50 years of age. The disease is caused by a genetic defect that is passed from parent to child in the Huntington gene. Having too many repeated elements in the gene sequence causes the disease and an increasing number of repeats leads to earlier onset and increased harshness of the condition.

The research team learned cerebrospinal fluid (CSF) from 30 individuals who carry the mutation that causes Huntington's disease, but who were not yet showing symptoms. They were able to identify modified levels of a molecule (also called microRNA) in individuals as much as twenty years before the expected onset of symptoms.

The most interesting discovery was that the levels of the microRNAs begin to increase many years before the individual shows symptoms and continue to increase as disease onset approaches. Clinical trials for new Huntington's disease treatments that may reduce the levels of the microRNAs suggest that these treatments may postpone the onset of the disease.

According to the researchers, these discoveries may represent a more effective way to tell whether Huntington's disease treatments may postpone the disease before it begins.  By evaluating microRNAs in the CSF, it may become feasible to perform these trials in people who are Huntington's disease gene carriers, but who do not yet show symptoms, by giving evidence for which trials may postpone onset and provide more healthy years of life.

These discoveries also suggest that other microRNAs may also be important markers of severity for other neurological diseases such as Parkinson's and Alzheimer's.

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