Lysosomal storage diseases
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Abstract
Lysosomes are subcellular organelles responsible for the physiologic
turnover of cell constituents. Lysosomal storage diseases describe a heterogeneous group
of dozens of rare inherited disorders characterized by the accumulation of undigested or
partially digested macromolecules, which ultimately results in cellular dysfunction and
clinical abnormalities. Several studies have explored different methods of treatment, but
only are promising and safe even though they are very expensive.
Description
Individually, lysosomal storage disorders are rare genetic diseases.
However, as a group, they are relatively common and represent an important health problem. Their early diagnosis is paramount since there are increasing possible therapies even though majority are still going through clinical trials.
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Adenegan Adesola Idowu. Lysosomal storage diseases [Electronic resource] : [presentation PowerPoint] / Adesola Idowu Adenegan. – Electronic data (19 slides). – Kharkiv, 2012.