therapeutic 1,748 words KG: ent-gene-f7979b3d
section:therapeuticskind:therapeutictopic:parkinsons
Contents

ATP13A2 (PARK9) Targeting for Parkinson's Disease

🔬 Protein Info
Gene SymbolPARK9
N-terminal regulatory regionCytoplasmic
Transmembrane domainsMembrane-spanning
ATP-binding domainCytoplasmic
C-terminal tailCytoplasmic
Pathogenic MechanismCellular Consequence
Lysosomal dysfunctionImpaired acidification, reduced cathepsin activity, lipofuscin accumulation
Autophagy blockadeAccumulation of autophagosomes, failed mitophagy
Metal dysregulationManganese and zinc accumulation, iron-induced oxidative stress
ER stressUnfolded protein response activation
Alpha-synuclein accumulationProtein aggregation and Lewy body formation
Dopaminergic neuron lossMotor and cognitive deficits
VectorPromoter
AAV9Synapsin
AAVrh.10CAG
AAV2Synapsin
DatabasesGeneCardsUniProtNCBI GeneHPASTRING

Knowledge Graph

Related Hypotheses (30)

Targeting Bacterial Curli Fibrils to Prevent α-Synuclein Cro
Score: 0.49
Trinucleotide Repeat Sequestration via CRISPR-Guided RNA Tar
Score: 0.48
Engineered Apolipoprotein E4-Neutralizing Shuttle Peptides
Score: 0.51
APOE4-Selective Lipid Nanoemulsion Therapy
Score: 0.49
APOE Isoform Conversion Therapy
Score: 0.44

Related Analyses (30)

Microglia-astrocyte crosstalk amplification loops in neurode
neurodegeneration · completed
APOE4 structural biology and therapeutic targeting strategie
neurodegeneration · completed
Autophagy-lysosome pathway convergence across neurodegenerat
neurodegeneration · completed
TDP-43 phase separation therapeutics for ALS-FTD
neurodegeneration · archived
Astrocyte reactivity subtypes in neurodegeneration
neurodegeneration · archived

Related Experiments (30)

Experiment: Multi-Ethnic PD GWAS
clinical · proposed · Score: 0.40
Synaptic Mitochondrial Resilience Enhancement for Parkinson'
validation · proposed · Score: 0.40
AAV Serotype Comparison for LRRK2 Knockdown in PD Gene Thera
validation · proposed · Score: 0.40
AAV-LRRK2 IND-Enabling Study Design
clinical · proposed · Score: 0.40
AAV Serotype Comparison for LRRK2 Knockdown in PD
validation · proposed · Score: 0.40

See Also (6)

UPenn Observational Research Repository (NCT04715399)
clinical · Pages share 3 hypotheses
remternetug-anti-amyloid-alzheimers
clinical_trial · Pages share 3 hypotheses
nct05508789
clinical_trial · Pages share 3 hypotheses
LX1001 Long-Term Follow-up (NCT05400330) - Gene Therapy
clinical · Pages share 3 hypotheses
LX1001 Phase 1/2 Trial (NCT03634007) - Gene Therapy for
clinical · Pages share 3 hypotheses

Knowledge Graph (2 edges)

ent-gene-f7979b3d data_in benchmark_ot_ad_answer_key:ATP13A2
benchmark_ot_ad_answer_key:ATP13A2 data_in ent-gene-f7979b3d