Fucosidosis is a lysosomal storage disorder, caused by mutations in the FUCA1 gene, resulting in impaired activity of alpha-L-fucosidase enzyme, leading to accumulation of complex sugars in the body, and eventually to death.
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Cell Therapy Approaches
Cell therapy utilizing hematopoietic stem cells has been associated with cell engraftment into the bone marrow and their differentiation into non-diseased blood cells, restoring functional alpha-L-fucosidase levels.