Validation of an AAV gene therapy designed to complement the loss of TDP‐43 splicing repression for ALS‐FTD

Description

Aswathy Peethambaran Mallika and Philip C. Wong report a gene therapy validation study for ALS-FTD, published in two thousand twenty-five. The work concerns TDP-43, a protein that normally represses faulty splicing of RNA. Loss of that function is thought to drive amyotrophic lateral sclerosis and frontotemporal dementia.

Earlier work used a fusion protein, called CTR, to replace the lost repression in young mice. Here the team tested a newer viral vector, AAV.PHP.eB, given by vein to adult mice. The vector reaches neurons in the brain and spinal cord efficiently. Treated animals were compared with controls receiving a harmless marker protein. The study checks whether this delivery route works well enough to move toward clinical use.

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Source: https://europepmc.org/articles/PMC11710014