Research ArticleLysosomal Storage Diseases

Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with mucopolysaccharidosis type VII

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Science Translational Medicine  26 Feb 2020:
Vol. 12, Issue 532, eaay8980
DOI: 10.1126/scitranslmed.aay8980

Article Information

vol. 12 no. 532

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication August 29, 2019
  • Accepted for publication January 28, 2020
  • .

Author Information

  1. Quoc-Hung Nguyen1,2,*,
  2. Russell G. Witt1,2,*,
  3. Bowen Wang1,2,
  4. Carlo Eikani1,2,
  5. Jeremy Shea1,3,
  6. Lucas K. Smith3,4,
  7. Gabrielle Boyle5,
  8. Jaclyn Cadaoas5,
  9. Renan Sper1,2,
  10. John D. MacKenzie6,
  11. Saul Villeda1,3 and
  12. Tippi C. MacKenzie1,2,7,
  1. 1Eli and Edythe Broad Center of Regeneration Medicine, University of California, San Francisco, San Francisco, CA 94143, USA.
  2. 2Department of Surgery, University of California, San Francisco, San Francisco, CA 94143, USA.
  3. 3Department of Anatomy, University of California, San Francisco, San Francisco, CA 94143, USA.
  4. 4Biomedical Sciences Graduate Program, University of California, San Francisco, San Francisco, CA 94143, USA.
  5. 5Ultragenyx Pharmaceutical Inc., Novato, CA 94949, USA.
  6. 6Department of Radiology, University of California, San Francisco, San Francisco, CA 94143, USA.
  7. 7Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, CA 94143, USA.
  1. Corresponding author. Email: tippi.mackenzie{at}ucsf.edu
  • * These authors contributed equally to this work.

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