disease 3,135 words KG: ent-dise-3e8e2e7c 2026-03-24
kind:diseasesection:diseasesstate:published
Contents

Sandhoff Disease

Disease Info
Point mutationsVarious missense and nonsense mutations that reduce or eliminate enzyme activity
Splice site mutationsMutations that disrupt normal RNA splicing
DeletionsLarger deletions that remove part or all of the gene
HexAA heterodimer of alpha and beta subunits (αβ)
HexBA homodimer of beta subunits (ββ)
HexSA homodimer of alpha subunits (αα)
Early signsWeakness, poor head control, decreased alertness
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (7)

Purinergic Signaling Polarization Control
Score: 0.55
Mechanosensitive Ion Channel Reprogramming
Score: 0.51
Lipid Droplet Dynamics as Phenotype Switches
Score: 0.46
Ganglioside Rebalancing Therapy
Score: 0.50
Sphingomyelin Synthase Activators for Raft Remodeling
Score: 0.47

Related Analyses (17)

Astrocyte reactivity subtypes in neurodegeneration
neurodegeneration · archived
TREM2 agonism vs antagonism in DAM microglia
neurodegeneration · failed
Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · archived
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · completed
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · archived

See Also (15)

Yoga Therapy for Neurodegeneration
therapeutic · Pages share 3 hypotheses
YAP/TEAD Pathway Modulators for Neurodegeneration
therapeutic · Pages share 3 hypotheses
Wnt Signaling Modulators for Neurodegeneration
therapeutic · Pages share 3 hypotheses
vitamin-d-therapy-neurodegeneration
therapeutic · Pages share 3 hypotheses
Vitamin B Complex Therapy for Neurodegeneration
therapeutic · Pages share 3 hypotheses