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An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
Giorgia Ceravolo1,2, Kristina Zhelcheska3, Violetta Squadrito4
1Department of Neuromuscular Disorders, Institute of Neurology, University College London (UCL), London, UK. g.ceravolo@ucl.ac.uk.
Leukodystrophies, rare white matter disorders, pose diagnostic challenges. Advances in genetic testing and emerging gene therapies offer new hope for treatment and clinical trial eligibility.
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Published on: September 29, 2014
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