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.
Researchers demonstrated the effectiveness of the direct delivery of gene therapy by delivering it into the brain of a mouse.
CTE is much more prevalent than was initially realised, and there is currently no therapy available. This new work is potentially ground-breaking as a means to remove the offending Tau phoshoprotein, said the lead researcher.
The disease which is caused by recurrent trauma in the central nervous system, such as those suffered by soldiers, atheletes 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-p-Tau antibody to the CNS. They showed that direct delivery of the AAVrh,10anti-p_tau directly into the hippocampus of brain-injured mice was associated with a significant reduction in p-Tau levels across the CNS.
They propose that doses could be scaled up and this strategy could be effective in humans as well.
Researchers demonstrated the effectiveness of the direct delivery of gene therapy by delivering it into the brain of a mouse.
CTE is much more prevalent than was initially realised, and there is currently no therapy available. This new work is potentially ground-breaking as a means to remove the offending Tau phoshoprotein, said the lead researcher.
The disease which is caused by recurrent trauma in the central nervous system, such as those suffered by soldiers, atheletes 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-p-Tau antibody to the CNS. They showed that direct delivery of the AAVrh,10anti-p_tau directly into the hippocampus of brain-injured mice was associated with a significant reduction in p-Tau levels across the CNS.
They propose that doses could be scaled up and this strategy could be effective in humans as well.