JAN 04, 2020 11:44 AM PST

Testing Gene Therapy as a Potential Treatment for CTE

WRITTEN BY: Carmen Leitch

Repeated trauma to the head, like a boxer, football player, or soldier might experience, appears to cause a degenerative disease called chronic traumatic encephalopathy (CTE). Researchers are still learning about the disease, and physical evidence connecting repeated head trauma to a disorder was discovered only recently, in 2002 by Dr. Bennet Omalu.

There is no specific symptom that has been conclusively linked to CTE, which can only be diagnosed by a post-mortem examination of the brain. But, symptoms may include problems thinking or carrying out tasks, depression, apathy, impulsive behavior, short-term memory loss, emotional instability, substance abuse, or suicidal thoughts.

Little is known about how the disease progresses or how to treat it. However, a protein called tau is known to build up in the brains of people with CTE, after they become chemically altered in a way that's called hyperphosphorylation. Tau proteins form in a pattern that's specific to CTE, and has also been detected at abnormally high levels in a study of people that had experienced repeated head trauma. That work suggested that high Tau levels may be a biomarker for the disease.

 New research reported in Human Gene Therapy has investigated whether gene therapy might be an effective therapeutic for CTE. Researchers delivered a viral vector (the typical carrier for gene therapy) directly to the brains of a mouse model of CTE. The scientists found that this approach significantly reduced the levels of a hyperphosphorylated form of Tau protein, called pTau.

"CTE is much more prevalent than was initially realized, and there is currently no therapy available," said Human Gene Therapy's Editor-in-Chief Terence R. Flotte, M.D., Celia and Isaac Haidak Professor of Medical Education and Dean, Provost. "This new work from the Crystal laboratory is potentially ground-breaking as a means to remove the offending Tau phoshoprotein."


Sources: AAAS/Eurekalert! via Mary Ann Liebert, Inc./Genetic Engineering News, Human Gene Therapy

About the Author
  • Experienced research scientist and technical expert with authorships on 28 peer-reviewed publications, traveler to over 60 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
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