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mouse protein expression lysosomal promoter activity neuraminidase storage disease enzyme tissue murine sequence level transcription factor binding c /ebpg analysis control globin model galactosialidosi region liver cathepsin result /ebpg mutation marrow brain probe patient study disorder fragment function locus transcriptional deficiency phenotype 5 hs 3 receptor transplantation genomic transcript effect development number 1995. fibroblast substrate product neuraminidase activity biochem kidney 1997. suzuki reporter lysosomal storage disorders finger regulation lysosomal storage animal lysosomal enzymes month family bone marrow transgene organization protein /cathepsin correction b-globin erythroid chapter meklf structure bone marrow transplantation mouse model hormone
