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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

Troy C. Lund,Lalitha Belur,Avery Huber,Hillary Mantone,Miles Smith,Kelly Podetz-Pedersen,Andrea Karlen,Elizabeth Braunlin,Nick Robinson,Hsing-Chen Tsai,R. Scott McIvor,Melissa Bonner

MOLECULAR GENETICS AND METABOLISM（2021）

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Key words
mucopolysaccharidosis type,hematopoietic stem cells,stem cells,lentiviral vector transduction,alpha-l-iduronidase
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