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
MOLECULAR GENETICS AND METABOLISM(2021)
Key words
mucopolysaccharidosis type,hematopoietic stem cells,stem cells,lentiviral vector transduction,alpha-l-iduronidase
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