Researchers have successfully used a noninvasive intranasal approach to deliver the gene for the enzyme that is deficient in the inherited lysosomal storage disease mucopolysaccharidosis type 1 (MPS 1) to the brains of an MPS mouse model, and have demonstrated the presence of the therapeutic enzyme throughout the mouse brains. Five months later, the treated mice did not exhibit the learning and navigation deficiencies seen in untreated mice, as reported in an article published in Human Gene Therapy.
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