NeurosciencebioRxivPreprint
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…·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.
3 citations