University of British Columbia researchers have developed a new test that allows physicians to gauge the effectiveness of a new therapy used to treat Huntington's disease.
The findings, published in
Scientific Reports, pave the way for the first human clinical trials a drug that targets the genetic cause of the disease,
Medical Xpress reports.
The gene-silencing therapy being tested by UBC researchers reduces the levels of a toxic brain protein that causes Huntington's.
"This is an important breakthrough for several promising gene silencing therapies in Huntington's disease that are now moving from the bench to the bedside," said Blair Leavitt of UBC's Centre for Molecular Medicine and Therapeutics and the Centre for Huntington Disease, who helped develop the test, in collaboration with colleagues from Mayo Clinic.
"We can move forward with these clinical trials and accurately measure whether our treatments are working."
Huntington's disease is a genetic disorder that affects the brain and gradually worsens, causing problems with coordination and movement, mental decline, and psychiatric issues.
The genetic mutation responsible for Huntington's produces a toxic form of a protein called huntingtin, which injures brain cells.
Several huntingtin-lowering therapies have shown promise in animal studies and are posed for early trials in human patients.
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