disease 2,151 words KG: ent-dise-6eeca971 2026-03-23
kind:diseasesection:diseasesstate:published
Contents

Gaucher Disease

Disease Info
N370S (p.Asn409Ser)The most frequent mutation, associated with Type 1 disease and residual enzyme activity. Homozygosity for N370S typically results in mild to moderate Type 1 disease.
L444P (p.Leu483Pro)Associated with neuronopathic disease when homozygous. This mutation results in severely reduced enzyme activity.
84insGA frameshift mutation common in Ashkenazi Jewish patients.
D409H (p.Asp441His)Associated with cardiovascular calcification and specific Type 3 phenotypes.
V433L (p.Val460Leu)A mutation associated with Type 3 disease in certain populations.
Mechanical effectsCell accumulation causes organ enlargement and bone marrow infiltration.
Inflammatory mediatorsGaucher cells secrete cytokines (IL-6, IL-10, TNF-α, CCL18) that contribute to systemic inflammation and bone pathology. [@pandey2022]
Cellular dysfunctionLipid accumulation disrupts normal macrophage function.
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (2)

Selective Acid Sphingomyelinase Modulation Therapy
Score: 0.65
Lysosomal Positioning Dynamics Modulation
Score: 0.43

Related Analyses (17)

Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · archived
Lipid raft composition changes in synaptic neurodegeneration
neurodegeneration · completed
TREM2 agonism vs antagonism in DAM microglia
neurodegeneration · failed
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · completed
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · archived

Related Experiments (1)

ER-Golgi Secretory Pathway Dysfunction in PD - Experiment De
clinical · proposed · Score: 0.40