Abigail E Hopkins

AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice.

C. Lorson, J. Marquez, E. Villalón, 2020, Biochemical and biophysical research communications.

AAV9-Mediated Delivery of miR-23a Reduces Disease Severity in Smn2B-/SMA Model Mice.

C. Lorson, A. Ebert, H. Rindt, 2019, Human molecular genetics.