Ex vivo lentiviral transduction of hematopoietic stem cells in mucopolysaccharidosis type II (MPS II) mice achieves high levels of systemic iduronate-2-sulfatase (IDS) enzyme activity and normalization of glycosaminoglycans (GAGs)

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Miles C. Smith, Lalitha Belur, Olivia Erlanson, Kelly Podetz-Pedersen, Andrea Karlen, Jessica McKenzie, Jenn Detellis, Khatuna Gagnidze, Geoff Parsons, Nick Robinson, Shelby Labarre, Gina Scarglia, Saumil Shah, Hsing-Chen Tsai, R. Scott McIvor, Melissa Bonner
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research