Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with mucopolysaccharidosis type VII-Reference-Cited by-同舟云学术

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

Published:2020-02-26 Issue:532 Volume:12 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Nguyen Quoc-Hung¹²^ORCID,Witt Russell G.¹²^ORCID,Wang Bowen¹²^ORCID,Eikani Carlo¹²,Shea Jeremy¹³^ORCID,Smith Lucas K.³⁴,Boyle Gabrielle⁵^ORCID,Cadaoas Jaclyn⁵,Sper Renan¹²^ORCID,MacKenzie John D.⁶^ORCID,Villeda Saul¹³,MacKenzie Tippi C.¹²⁷^ORCID

Affiliation:

1. Eli and Edythe Broad Center of Regeneration Medicine, University of California, San Francisco, San Francisco, CA 94143, USA.

2. Department of Surgery, University of California, San Francisco, San Francisco, CA 94143, USA.

3. Department of Anatomy, University of California, San Francisco, San Francisco, CA 94143, USA.

4. Biomedical Sciences Graduate Program, University of California, San Francisco, San Francisco, CA 94143, USA.

5. Ultragenyx Pharmaceutical Inc., Novato, CA 94949, USA.

6. Department of Radiology, University of California, San Francisco, San Francisco, CA 94143, USA.

7. Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, CA 94143, USA.

Abstract

In utero therapy with recombinant enzyme or hematopoietic stem cells in MPS7 mice induces tolerance to missing enzyme and corrects brain microglia.

Funder

National Institutes of Health

California Institute for Regenerative Medicine

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference72 articles.

1. Beta glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

2. The many faces of hydrops

3. Clinical course of sly syndrome (mucopolysaccharidosis type VII)

4. Lysosomal storage disease as an etiology of nonimmune hydrops

5. Emptying the stores: lysosomal diseases and therapeutic strategies

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