Lysosomal Storage Diseases

2010

Lysosomal storage disease is an inherited genetic defect causing a deficiency or malfunction of one of the enzymes in the lysosomes. The disease is progressive in nature and may cause multi-organic, irreversible damage that can be seriously debilitating and life-threatening. One treatment option is enzyme replacement therapy with enzymes produced by recombinant technology. The 3d animation film shows the effect of this technology.

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