Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

by Li Ou, Michael J. Przybilla, Brenda L. Koniar, Chester B. Whitley
Published 2016-09-01

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

by Michael J. Przybilla, Christine Stewart, Timothy W. Carlson, Li Ou, Brenda L. Koniar, Rohini Sidhu, Pamela J. Kell, Xuntian Jiang, Jeanine R. Jarnes, M. Gerard O'Sullivan, Chester B. Whitley
Published 2021-06-01