Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by a deficiency of the enzyme arylsulfatase A, which results in accumulation of sphingolipids, eventually destroying the myelin sheath in the nervous system.
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Cell Therapy Approaches
In patients with enzymatic abnormalities and metabolic storage disorders, cell therapy enables synthesis of normal functional enzymes that can circulate and improve cellular functions of the native tissues. Cell therapy based upon bone marrow and bone marrow-derived stem cell transplantation can potentially reduce or halt disease progression in the central nervous system.