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Development of Enzyme Replacement and Pharmacological Chaperone Approaches for Therapy of Metachromatic Leukodystrophy
(2015-01-06)
Metachromatic leukodystrophy (MLD) is a monogenic, autosomally recessively inherited lysosomal storage disease. Certain mutations of the arylsulfatase A (ASA) gene cause misfolding or absence of ASA. Physiologically ASA ...