disease 1,464 words KG: ent-dise-a368f850 2026-03-19
kind:diseasesection:diseasesstate:published
Contents

Kufs Disease

Disease Info
Typical OnsetMean onset around 28 years (range: teens to adulthood)
GeneCLN6 (MIM 606725)
Chromosomal Location15q23
Inheritance PatternAutosomal recessive
Protein FunctionCLN6 is a transmembrane protein located in the endoplasmic reticulum, involved in lysosomal function and lipid metabolism <sup>[4]</sup>
DNAJC5Autosomal dominant form
CLN6Autosomal recessive form
GRN (Progranulin)Some cases of frontotemporal dementia-like presentation
CSTB (Cystatin B)Related to progressive myoclonus epilepsy
Neuronal LossProgressive death of neurons in the cerebral cortex, cerebellum, and brainstem
Lipopigment AccumulationStorage material composed of ceroid and lipofuscin within lysosomes
AtrophyCerebral and cerebellar atrophy visible on MRI
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (7)

Lysosomal Calcium Channel Modulation Therapy
Score: 0.49
Lysosomal Enzyme Trafficking Correction
Score: 0.49
APOE Isoform Expression Across Glial Subtypes
Score: 0.48
Lysosomal Membrane Repair Enhancement
Score: 0.45
Microbial Metabolite-Mediated α-Synuclein Disaggregation
Score: 0.45

Related Analyses (16)

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
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived