Ex Vivo Lentiviral Transduction Of Hematopoietic Stem Cells In Mucopolysaccharidosis Type Ii (Mps Ii) Mice Achieves High Levels Of Systemic Iduronate-2-Sulfatase (Ids) Enzyme Activity And Normalization Of Glycosaminoglycans (Gags)
MOLECULAR GENETICS AND METABOLISM(2021)
关键词
mucopolysaccharidosis type ii,glycosaminoglycans,lentiviral transduction,stem cells,hematopoietic stem cells
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