Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice-Reference-Cited by-同舟云学术

Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice

Published:2020-12-15 Issue:1 Volume:18 Page:214-227
ISSN:1543-8384
Container-title:Molecular Pharmaceutics
language:en
Short-container-title:Mol. Pharmaceutics

Author:

Wang Feng¹²,Moen Derek R.³,Sauni Chelsee³,Kan Shih-hsin¹⁴,Li Shan¹²,Le Steven Q.¹⁵,Lomenick Brett⁶,Zhang Xiaoyi¹,Ekins Sean³^ORCID,Singamsetty Srikanth³,Wood Jill³,Dickson Patricia I.¹⁵,Chou Tsui-Fen¹²⁶^ORCID

Affiliation:

1. Division of Medical Genetics, Department of Pediatrics, Harbor-UCLA Medical Center and The Lundquist Institute, Torrance, California 90502, United States

2. Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California 91125, United States

3. Phoenix Nest Inc., Brooklyn, New York 11232, United States

4. Research Administration, CHOC Children’s Hospital, Orange, California 92868, United States

5. Department of Pediatrics, Washington University in St. Louis, St. Louis, Missouri 63110, United States

6. Proteome Exploration Laboratory, Beckman Institute, California Institute of Technology, Pasadena, California 91125, United States

Funder

National Institute of Neurological Disorders and Stroke

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Pharmaceutical Science,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/acs.molpharmaceut.0c00831

Reference82 articles.

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4. Mucopolysaccharide diseases: A complex interplay between neuroinflammation, microglial activation and adaptive immunity

5. Sanfilippo syndrome: A mini-review

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