Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells

Human Gene Therapy, Ahead of Print.

Source: Human Gene Therapy - November 23, 2022 Category: Genetics & Stem Cells Authors: Miles C. Smith Lalitha R. Belur Andrea D. Karlen Olivia Erlanson Kelly M. Podetz-Pedersen Jessica McKenzie Jenn Detellis Khatuna Gagnidze Geoffrey Parsons Nicholas Robinson Shelby Labarre Saumil Shah Justin Furcich Troy C. Lund Hsing-Chen Tsai R. Scott Mc Source Type: research