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Research ArticleLysosomal Storage Diseases
Replacing the Enzyme α-l-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I
- Ashley D. Dierenfeld1,
- Michael F. McEntee2,
- Carole A. Vogler3,
- Charles H. Vite4,
- Agnes H. Chen5,
- Merry Passage5,
- Steven Le5,
- Sahil Shah5,
- Jackie K. Jens1,
- Elizabeth M. Snella1,
- Karen L. Kline6,
- Jennifer D. Parkes6,
- Wendy A. Ware6,
- Lori E. Moran6,
- Amanda J. Fales-Williams7,
- Jane A. Wengert6,
- R. David Whitley6,
- Daniel M. Betts6,
- Amy M. Boal6,
- Elizabeth A. Riedesel6,
- William Gross6,
- N. Matthew Ellinwood1,6,* and
- Patricia I. Dickson5
- 1Department of Animal Science and Center for Integrated Animal Genomics, Iowa State University, Ames, IA 50011–3150, USA.
- 2Department of Pathobiology, College of Veterinary Medicine, University of Tennessee, Knoxville, TN 37996, USA.
- 3Department of Pathology, St. Louis University, St. Louis, MO 63104, USA.
- 4Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104–6010, USA.
- 5Los Angeles Biomedical Research Institute at Harbor–UCLA Medical Center, and the Department of Pediatrics at Harbor-UCLA Medical Center, Torrance, CA 90502, USA.
- 6Department of Veterinary Clinical Sciences, College of Veterinary Medicine, Iowa State University, Ames, IA 50011–1250, USA.
- 7Veterinary Pathology, College of Veterinary Medicine, Iowa State University, Ames, IA 50011–1250, USA.
- *To whom correspondence should be addressed. E-mail: mellinwo{at}iastate.edu
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Science Translational Medicine 01 Dec 2010:
Vol. 2, Issue 60, pp. 60ra89
DOI: 10.1126/scitranslmed.3001380
Vol. 2, Issue 60, pp. 60ra89
DOI: 10.1126/scitranslmed.3001380
Ashley D. Dierenfeld
Michael F. McEntee
Carole A. Vogler
Charles H. Vite
Agnes H. Chen
Merry Passage
Steven Le
Sahil Shah
Jackie K. Jens
Elizabeth M. Snella
Karen L. Kline
Jennifer D. Parkes
Wendy A. Ware
Lori E. Moran
Amanda J. Fales-Williams
Jane A. Wengert
R. David Whitley
Daniel M. Betts
Amy M. Boal
Elizabeth A. Riedesel
William Gross
N. Matthew Ellinwood
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