Retromer Combinatorials for Gene-Therapy Across a Spectrum of Neurological Diseases
Yasir H. Qureshi, Vivek M. Patel, Suvarnambiga Kannan, Samuel D. Waksal, Gregory A. Petsko, Scott A. Small
The paper tests combinatorial gene therapy for retromer-related neurological diseases by expressing VPS35 with VPS26 paralogs in neurons, showing synergistic effects on retromer function and Sorl1 levels.
bioRxiv · Sep 3, 2020 · PreprintNeuroscience