disease 712 words KG: ent-dise-226a4c9b 2026-03-21
kind:diseasesection:diseasesstate:published
Contents

Huntington's Disease-Like 2 (HDL2)

Disease Info
InheritanceAutosomal dominant
Gene*JPH3* (junctophilin-3)
Mutation TypeCGG trinucleotide repeat expansion in the 5' UTR
Chromosomal Location16q24.3
Normal Repeat6-27 CGG repeats
Pathogenic Repeat41-58+ CGG repeats
RNA-mediated toxicityExpanded CGG repeats may form toxic RNA structures that sequester essential RNA-binding proteins
Loss of JPH3 functionJunctophilin-3 is involved in calcium homeostasis at the endoplasmic reticulum-mitochondria contact sites
Age of onsetTypically 30-50 years, but can range from early 20s to late 60s
Disease duration10-20 years from symptom onset to death
ProgressionGradual decline in motor and cognitive function
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph

Related Hypotheses (3)

Hippocampal CA3-CA1 circuit rescue via neurogenesis and syna
Score: 0.68
Trinucleotide Repeat Sequestration via CRISPR-Guided RNA Tar
Score: 0.48
Enteric Nervous System Prion-Like Propagation Blockade
Score: 0.44

Related Analyses (16)

What are the mechanisms by which gut microbiome dysbiosis in
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
Protein aggregation cross-seeding across neurodegenerative d
neurodegeneration · archived

Related Experiments (2)

Epigenetic Dysregulation in Huntington's Disease — Therapeut
validation · proposed · Score: 0.40
Microglial Contributions to Huntington's Disease Pathogenesi
validation · proposed · Score: 0.40