Metachromatic leukodystrophy and transplantation: remyelination, no cross-correction.,Annals of Clinical and Translational Neurology

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Metachromatic leukodystrophy and transplantation: remyelination, no cross-correction.
Annals of Clinical and Translational Neurology ( IF 5.3 ) Pub Date : 2020-01-22 , DOI: 10.1002/acn3.50975
Nicole I Wolf ₁ , Marjolein Breur _{1,

2} , Bonnie Plug _{1,

2} , Shanice Beerepoot _{1,

3} , Aimee S R Westerveld _{1,

2} , Diane F van Rappard ₁ , Sharon I de Vries ₄ , Maarten H P Kole _{4,

5} , Adeline Vanderver _{6,

7} , Marjo S van der Knaap _{1,

8} , Caroline A Lindemans _{9,

10} , Peter M van Hasselt ₁₁ , Jaap J Boelens ₉ , Ulrich Matzner ₁₂ , Volkmar Gieselmann ₁₂ , Marianna Bugiani _{1,

2}

Affiliation

Department of Child Neurology, Center for Childhood White Matter Diseases, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, and Amsterdam Neuroscience, Amsterdam, The Netherlands.
Department of Pathology, Amsterdam Neuroscience, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.
Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Axonal Signaling, Netherlands Institute for Neuroscience, Amsterdam, The Netherlands.
Cell Biology Faculty of Science, Utrecht University, Utrecht, The Netherlands.
Division of Neurology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, Pennsylvania.
Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam, The Netherlands.
Department of Pediatrics, University Medical Center Utrecht, Utrecht, The Netherlands.
Pediatric Blood and Marrow Transplantation Program, Princess Maxima Center, Utrecht, The Netherlands.
Department of Metabolic Diseases, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, The Netherlands.
Institute of Biochemistry and Molecular Biology, Rheinische Friedrich-Wilhelms University, Bonn, Germany.

In metachromatic leukodystrophy, a lysosomal storage disorder due to decreased arylsulfatase A activity, hematopoietic stem cell transplantation may stop brain demyelination and allow remyelination, thereby halting white matter degeneration. This is the first study to define the effects and therapeutic mechanisms of hematopoietic stem cell transplantation on brain tissue of transplanted metachromatic leukodystrophy patients.

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