Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy
Mayra F. Garcia-Rivera, Leah E. Colvin-Wanshura, Matthew S. Nelson, Zhenhong Nan, Shaukat A. Khan, Tyson B. Rogers, Indrani Maitra, Walter C. Low, Pankaj Gupta
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