Busulfan conditioning allows high engraftment of human genome edited hematopoietic stem cells and improved central nervous system correction in a mucopolysaccharidosis type I mouse model

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Edina Poletto, Pasqualina Colella, Adam Sheikali, Guilherme Baldo, Natalia Gomez-Ospina
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research