Publications by authors named "Emma Burkard"
Cell type-specific gene therapy confers protection against motor neuron disease caused by a TFG variant.
Proc Natl Acad Sci U S A
November 2024
Article Synopsis
- Inherited motor neuron diseases (MND) like hereditary spastic paraplegias (HSP) involve the death or dysfunction of nerve cells that control muscle activity, but genetic variants may also affect other supportive cells.
- Researchers used a rat model with a specific TFG mutation that mimics human HSP symptoms, such as motor deficits and spasticity, to investigate the source of axonopathy in the corticospinal tract.
- Results showed that introducing the normal TFG gene into specific neurons significantly improved motor function, while targeting supportive glial cells did not yield similar benefits, highlighting the importance of neuron-focused therapies for TFG-related HSP.