Lymphocyte Gene Therapy for Mild Hunter Syndrome 
Fig. 4 - Decreased Radiosulfate Accumulation By Co-Cultivation 
With L2SN-Transduced LCLmps 
These results demonstrate that the high-level IDS enzyme levels of LCL not only correct 
metabolism in the LCL, but also mediate intercellular cross-correction of lysosomal metabolism in 
co-cultivated, untransduced Hunter fibroblasts. These results are significant in the development 
of gene therapy for Hunter syndrome because they demonstrate that recombinant enzyme 
expressed from a transduced lvmphohematopoietic cell is capable of gaining access to cells of a 
distinct type and participating in the metabolism of glycosaminoglycans. In the transfer and 
expression of IDS and other genes in cells of the circulatory system, such intercellular cross 
correction is exactly what will be needed for this approach to be effective in correcting symptoms 
Recombinant DNA Research, Volume 20 
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