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

• DOI: 10.1126/scitranslmed.aay8980
• Publication Date: 2020-02-27T00:05:23Z
• Authors: Quoc-Hung Nguyen; Russell G. Witt; Bowen Wang; Carlo Eikani; Jeremy Shea; Lucas K. Smith; Gabrielle Boyle; Jaclyn Cadaoas; Renan Sper; John D. MacKenzie; Saul Villeda; Tippi C. MacKenzie
• Keywords: Mucopolysaccharidosis; Mucopolysaccharidosis type I

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