Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.

@article{Tong2017GuanidinylatedNC,
  title={Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain.},
  author={Wenyong Tong and Chrissa A. Dwyer and Bryan Edward Thacker and Charles A. Glass and Jillian R. Brown and Kristina M. Hamill and Kelley W Moremen and St{\'e}phane Sarrazin and Philip L. S. M. Gordts and Lara E. Dozier and Gentry N Patrick and Yitzhak Tor and Jeffrey D Esko},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2017},
  volume={25 12},
  pages={
          2743-2752
        }
}
Iduronidase (IDUA)-deficient mice accumulate glycosaminoglycans in cells and tissues and exhibit many of the same neuropathological symptoms of patients suffering from Mucopolysaccharidosis I. Intravenous enzyme-replacement therapy for Mucopolysaccharidosis I ameliorates glycosaminoglycan storage and many of the somatic aspects of the disease but fails to treat neurological symptoms due to poor transport across the blood-brain barrier. In this study, we examined the delivery of IDUA conjugated… CONTINUE READING