Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood‐onset beta‐mannosidosis-Reference-Cited by-同舟云学术

Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood‐onset beta‐mannosidosis

Published:2019-05-21 Issue:7 Volume:7 Page:
ISSN:2324-9269
Container-title:Molecular Genetics & Genomic Medicine
language:en
Short-container-title:Mol Genet Genomic Med

Author:

Lund Troy C.¹^ORCID,Miller Weston P.²,Eisengart Julie B.³^ORCID,Simmons Katrina⁴,Pollard Laura⁵,Renaud Deborah L.⁶,Wenger David A.⁷,Patterson Marc C.⁸,Orchard Paul J¹

Affiliation:

1. Division of Pediatric Blood and Marrow Transplant University of Minnesota Minneapolis Minnesota

2. Sangamo Therapeutics Richmond California

3. Division of Clinical Behavioral Neuroscience, Department of Pediatrics University of Minnesota Minneapolis Minnesota

4. Sanofi, Rare Disease Division Sanofi Genzyme US Bridgewater New Jersey

5. Biochemical Genetics Laboratory Greenwood Genetic Center Greenwood South Carolina

6. Department of Neurology, Department of Clinical Genomics, Department of Pediatrics Mayo Clinic Rochester Minnesota

7. Lysosomal Diseases Testing Laboratory, Department of Neurology Sidney Kimmel Medical College, Thomas Jefferson University Philadelphia Pennsylvania

8. Division of Child and Adolescent Neurology Mayo Clinic Rochester Minnesota

Publisher

Wiley

Subject

Genetics(clinical),Genetics,Molecular Biology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mgg3.712

Reference36 articles.

1. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

2. Human -Mannosidase cDNA Characterization and First Identification of a Mutation Associated with Human -Mannosidosis

3. Fludarabine and Exposure-Targeted Busulfan Compares Favorably with Busulfan/Cyclophosphamide-Based Regimens in Pediatric Hematopoietic Cell Transplantation: Maintaining Efficacy with Less Toxicity

4. Variable clinical presentation of lysosomal β-mannosidosis in patients with null mutations

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