Effect of supraphysiological alpha-L-iduronidase (IDUA) expression on skeletal manifestations in mucopolysaccharidosis type I (MPS I) mice following ex vivo lentiviral vector transduction of hematopoietic stem cells
Titel:
Effect of supraphysiological alpha-L-iduronidase (IDUA) expression on skeletal manifestations in mucopolysaccharidosis type I (MPS I) mice following ex vivo lentiviral vector transduction of hematopoietic stem cells
Auteur:
Lund, Troy C. Belur, Lalitha Huber, Avery Mantone, Hillary Smith, Miles Podetz-Pedersen, Kelly Karlen, Andrea Braunlin, Elizabeth Robinson, Nick Tsai, Hsing-Chen McIvor, R. Scott Bonner, Melissa