disease 2,264 words KG: ent-dise-0ac66fce 2026-03-22
kind:diseasesection:diseasesstate:published
Contents

CARASIL (Cerebral Autosomal Recessive Arteriopathy with Subcortical Infarcts and Leukoencephalopathy)

Disease Info
PrevalenceUnknown, but estimated to be substantially rarer than](/diseases/cadasil) (which has an estimated prevalence of 2-5 per 100,000)
Geographic distributionThe majority of confirmed cases have been reported in **Japan** and **China**, though cases have also been identified in Turkey, Pakistan, Portugal, Spain, Romania, India, and other countries
Signal peptideTargets the protein for secretion
IGFBP domainInsulin-like growth factor binding protein domain
Serine protease domainCatalytic trypsin-like domain responsible for proteolytic activity
PDZ domainMediates protein-protein interactions and oligomerization
Nonsense mutationsCreating premature stop codons
Frameshift mutationsResulting in truncated, non-functional protein
Splice-site mutationsDisrupting mRNA processing
Heterozygous carriersHistorically considered asymptomatic, but now recognized to develop cerebral small vessel disease in some cases, typically with later onset (50s-60s) and without alopecia or spondylosis
Substrate cleavageHTRA1 degrades TGF-beta propeptides (latency-associated peptide), mature TGF-beta ligands, and downstream effectors
Extracellular matrix regulationHTRA1 cleaves fibronectin, vitronectin, decorin, fibromodulin, and other extracellular matrix (ECM) proteins, maintaining normal matrix turnover
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (8)

Bacterial Enzyme-Mediated Dopamine Precursor Synthesis
Score: 0.36
Purinergic Signaling Polarization Control
Score: 0.55
Mechanosensitive Ion Channel Reprogramming
Score: 0.51
Lipid Droplet Dynamics as Phenotype Switches
Score: 0.46
Dual-Domain Antibodies with Engineered Fc-FcRn Affinity Modu
Score: 0.57

Related Analyses (6)

Astrocyte reactivity subtypes in neurodegeneration
neurodegeneration · archived
Blood-brain barrier transport mechanisms for antibody therap
neurodegeneration · archived
What are the mechanisms by which gut microbiome dysbiosis in
neurodegeneration · archived
Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · archived
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived

Related Experiments (3)

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

See Also (15)

Yoga Therapy for Neurodegeneration
therapeutic · Pages share 4 hypotheses
YAP/TEAD Pathway Modulators for Neurodegeneration
therapeutic · Pages share 4 hypotheses
Wnt Signaling Modulators for Neurodegeneration
therapeutic · Pages share 4 hypotheses
vitamin-d-therapy-neurodegeneration
therapeutic · Pages share 4 hypotheses
Vitamin B Complex Therapy for Neurodegeneration
therapeutic · Pages share 4 hypotheses