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Sphingomyelin phosphodiesterase 1, acid lysosomal (SMPD1)
- Target
- SMPD1
- Molecular classification
- Enzyme, Hydrolase, Phosphodiesterase
01
Overview
Acid sphingomyelinase (SMPD1) is a lysosomal enzyme that catalyzes the hydrolysis of sphingomyelin into ceramide and phosphorylcholine. This reaction is crucial for lipid metabolism and cellular homeostasis. Ceramide produced by this process acts as a bioactive signaling molecule involved in cell differentiation, apoptosis, proliferation, and response to stress. Deficiency or mutations in SMPD1 lead to Niemann-Pick disease types A and B, characterized by the accumulation of sphingomyelin in various tissues and organs.
- Other names
- Acid sphingomyelinaseASMASM_HUMAN
02
Mechanism of action
Hydrolyzes sphingomyelin into ceramide and phosphorylcholine.
03
Biological functions
Lipid metabolismCell signalingApoptosis regulationCell proliferation regulation
04
Disease associations
Niemann-Pick disease type ANiemann-Pick disease type BLysosomal storage disorder
05
Safety considerations
Potential for off-target effects in enzyme replacement therapy or gene therapy.Immune response to enzyme replacement therapy.
06
Biomarkers
SMPD1 activity levelsSphingomyelin levelsCeramide levels
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