Uptake and lysosomal delivery of recombinant human alpha-n-acetylglucosamine-6-sulfatase to mucopolysaccharidosis IIID fibroblasts

  • Derek R. Moen, Xiaoyi Zhang, Kan Shin-hsin, Jill Wood, Sean Ekins, Tsui-Fen Chou, Patricia Dickson
  • Molecular Genetics and Metabolism, February 2016, Elsevier
  • DOI: 10.1016/j.ymgme.2015.12.365

MPSIIID enzyme replacement therapy

What is it about?

This poster describes the groups work to develop an enzyme replacement for Sanfilippo IIID (MPS IIID).

Why is it important?

There is no treatment for this disease. An ERT would be the first treatment developed.

Perspectives

Dr Sean Ekins
Collaborations in Chemistry

This work has been funded by an NINDS STTR. This poster describes the initial work from a phase I grant.

Read Publication

http://dx.doi.org/10.1016/j.ymgme.2015.12.365

The following have contributed to this page: Dr Sean Ekins