pharmacological implications of Lysosomal degradative enzymes

Expert Solution

Cellular degradation occurs in a controlled manner inside the organelles named as lysosomes by various processes like autophagy, endocytosis and phagocytosis. The cells ingest the microorganisms and other Nano particles (membrane vesicles) produced by the pathogen. Such Nano particles including the endosomes and phagosomes are delivered to the lysosomes which are then degraded by the lysosomal enzymes. Cell hemostasis is adversely affected by dysfunction of the lysosomes. Main cause of lysosomal dysfunction may be genetic deficiencies and mainly affects the central nervous system and the peripheral organs. In lysosomal deficiency the substrates accumulates in huge quantities which can impair the functioning of vital organs like brain and liver. Neurodegenerative conditions, ageing and certain infectious diseases can occur as a result of lysosomal malfunctioning.

Lysosomal Enzyme replacement therapy has proved to an important pharmacological measure in treating lysosomal disorders. Despite of its effectiveness, there are several short comings too like serious side effects, development of resistance, low therapeutic outcome as a result of sub optimal delivery through out the body.

Nightingale answered 1 year ago

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