UPSC Articles
Lysosomal Storage Disorders (LSD)
Part of: GS Prelims and GS-II- Health
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- The lysosomes are primary digestive units within cells. Their function is to break down complex components into simpler ones. They degrade complex components such as proteins (substrates) into simpler components
- When this process does not take place due to enzyme deficiency, the toxic substrates begin to accumulate in the cells. That is why these diseases are called “storage diseases”.
- Therefore, Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.
- Most lysosomal storage disorders are inherited in an autosomal recessive manner.
- There are nearly 50 of these disorders altogether, and they may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system
- There is currently no approved treatment for many lysosomal storage diseases.
