Carboxy-terminal blockade of sortilin binding enhances progranulin gene therapy, a potential treatment for frontotemporal dementia
Shreya N. Kashyap, Stephanie N. Fox, Katherine I. Wilson, Charles Murchison, Yohannes Abere Ambaw, Tobias C. Walther, Robert V. Farese, Andrew E. Arrant, Erik D. Roberson
Blocking the carboxy terminus of progranulin enhances gene therapy efficacy in progranulin-deficient mice by reducing sortilin interactions and improving biomarker outcomes.
bioRxiv · Sep 15, 2024 · PreprintNeuroscience