Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

Author:

Gupta Ashish O1,Raymond Gerald2,Pierpont Elizabeth I3ORCID,Kemp Stephan4,McIvor R Scott5,Rayannavar Arpana6,Miller Bradley6,Lund Troy C1,Orchard Paul J1ORCID

Affiliation:

1. Division of Pediatric Blood and Marrow Transplant and Cellular Therapies, University of Minnesota, Minneapolis, Minnesota, USA

2. Division of Neurogenetics and the Moser Center for Leukodystrophies, Kennedy Krieger Institute, Johns Hopkins University, Baltimore, MD, USA

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

4. Laboratory Genetic Metabolic Diseases, Department of Clinical Chemistry, Amsterdam UMC - University of Amsterdam, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam Neuroscience, Amsterdam, The Netherlands

5. Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA

6. Division of Pediatric Endocrinology, University of Minnesota, Minneapolis, Minnesota, USA

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology

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