Study suggests feasibility of treating encephalopathy with gene therapy

A new study has highlighted that it is feasible to use gene therapy for treating chronic traumatic encephalopathy (CTE) which is a progressive neurodegenerative disorder.

The study was published in the journal - Huma Gene Therapy.

Researchers demonstrated the effectiveness of the direct delivery of gene therapy by delivering it into the brain of a mouse.

Ronald Crystal and colleagues from Weill Cornell Medical College, New York, NY, coauthored the article entitled 'Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy'.

"CTE is much more prevalent than was initially realized, and there is currently no therapy available. This new work from the Crystal laboratory is potentially ground-breaking as a means to remove the offending Tau phoshoprotein," said the lead researcher Terence R. Flotte.

The disease which is caused by recurrent traumas in the central nervous system, such as those suffered by soldiers, athletes in contact sports, and in accidents, is currently incurable.

Inflammation results in the accumulation of hyperphosphorylated forms of Tau protein (pTau). Researchers developed an adeno-associated virus (AAV) vector to deliver an anti-pTau antibody to the (CNS). They showed that direct delivery of the AAVrh.10anti-pTau directly into the hippocampus of brain-injured mice was associated with a significant reduction in pTau levels across the CNS.

They propose that doses could be scaled up and this strategy could be effective in humans as well.