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Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy |
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| Authors: | Yonghong Chen Shujuan Zheng Luis Tecedor Beverly L. Davidson |
| Affiliation: | 1. Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA;2. Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA |
| Abstract: | |
| Keywords: | lysosomal storage disease protein engineering MPS gene therapy |
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