New gene therapy tools for brain and spine disorders
Image credit: nci via Unsplash
Scientists have developed specialized gene delivery “trucks” designed to target cells in the brain and spinal cord, offering a promising new approach for treating neurological diseases. As reported in the study published in Cell Reports, these engineered systems are capable of crossing the blood-brain barrier and delivering genetic material with precision. Researchers believe this breakthrough could pave the way for more effective therapies for conditions like Alzheimer’s, Parkinson’s, and spinal cord disorders.
Source
Kussick, E., Johansen, N., Taskin, N., Chowdhury, A., Quinlan, M. A., Fraser, A., Clark, A. G., Wynalda, B., Martinez, R., Groce, E. L., Reding, M., Liang, E., Shulga, L., Huang, C., Casper, T., Clark, M., Ho, W., Gao, Y., van Velthoven, C. T. J., Sobieski, C., … Daigle, T. L. (2025). Enhancer AAVs for targeting spinal motor neurons and descending motor pathways in rodents and macaque. bioRxiv : the preprint server for biology, 2024.07.30.605864. https://doi.org/10.1101/2024.07.30.605864
Additional Reading
https://www.washingtonpost.com/science/2025/05/28/gene-delivery-brain-diseases/
https://alleninstitute.org/news/scientists-can-now-target-the-cells-at-the-center-of-als/