Cell type-specific gene therapy confers protection against motor neuron disease caused by a TFG variant

Audhya, PhD, A. Cell Type-Specific Gene Therapy Confers Protection Against Motor Neuron Disease Caused by a TFG Variant.
Lettman, M. M., Mendina, C. A., Burkard, E., Alvin, J. R., Zhu, Y., Coon, J. J., & Audhya, A. (2024). Cell type-specific gene therapy confers protection against motor neuron disease caused by a TFG variant. Proceedings of the National Academy of Sciences of the United States of America, 121(47), e2410996121. https://doi.org/10.1073/pnas.2410996121