Protein Misfolding in Lysosomal Storage Disorders:Molecular Pathophysiology and Therapeutic Perspect

来源 :2011第二届国际神经科技大会 | 被引量 : 0次 | 上传用户:hnkfxwj
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  Lysosomes are cytoplasmic organelles harbouring over 100 hydrolytic enzymes involved in the degradation of biological macromolecules.Any failure in folding, maturation, lysosomal targeting, or function of one or more lysosomal enzymes can result in the progressive metabolic disorders called lysosomal storage diseases (LSD) because of the massive accumulation of the undegraded substrates of the deficient enzymes in the lysosomes.Recent studies showed that many neurological forms of lysosomal disorders are caused by misfolding and aggregation of mutant enzymes in the patient cells.
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