therapeutic 1,690 words KG: ent-gene-694200ea
Contents

gba-gene-therapy

🔬 Protein Info
Gene Symbolent-gene-694200ea
Active siteD237, E235
Mutation TypeExamples
Severe (null)L444P, 84insGG
ModerateN370S, D409H
Risk modifiersE326K, T369M
SerotypeTropism
AAV9Neurons, astrocytes
AAVrh.10Neurons
AAV-PHP.BNeurons
AAV2Neurons
ElementFunction
PromoterSynapsin (neuron-specific), CAG (strong ubiquitous)
IntronImproves expression stability
GBA cDNAWild-type glucocerebrosidase
PolyAAAV2 ITR-flanked
KG Connections2 knowledge graph edges
DatabasesGeneCardsUniProtNCBI GeneHPASTRING

Knowledge Graph

Related Hypotheses (30)

Bacterial Enzyme-Mediated Dopamine Precursor Synthesis
Score: 0.36
Chaperone-Mediated APOE4 Refolding Enhancement
Score: 0.48
Smartphone-Detected Motor Variability Correction
Score: 0.56
Microbial Metabolite-Mediated α-Synuclein Disaggregation
Score: 0.45
Enteric Nervous System Prion-Like Propagation Blockade
Score: 0.44

Related Analyses (23)

Does TFEB activation directly restore mutant GBA enzyme acti
molecular biology · failed
What is the therapeutic window between GCS inhibition effica
neurodegeneration · failed
GBA-Synuclein Loop: Therapeutic Strategies for Parkinson's D
neurodegeneration · completed
TDP-43 phase separation therapeutics for ALS-FTD
neurodegeneration · archived
APOE4 structural biology and therapeutic targeting strategie
neurodegeneration · archived

Related Experiments (17)

Alpha-Synuclein Aggregation Triggers — Sporadic PD Initiatio
clinical · proposed · Score: 0.40
Autophagy Enhancement Drug Screening for Neurodegeneration
clinical · proposed · Score: 0.40
Genetic Risk Modifiers in DLB Phenotype
clinical · proposed · Score: 0.40
DLB Treatment Response Biomarkers — Predicting Cholinesteras
clinical · proposed · Score: 0.40
DNA Damage Repair Deficiency Validation Study in Parkinson's
clinical · proposed · Score: 0.40

Knowledge Graph (2 edges)

ent-gene-694200ea data_in benchmark_ot_ad_answer_key:GBA
benchmark_ot_ad_answer_key:GBA data_in ent-gene-694200ea