| Gene Symbol | AAV |
| Full Name | LRRK2 Gene Therapy IND-Enabling Study Design |
| GeneCards | AAV |
| Human Protein Atlas | AAV |
| Efficacy threshold | Based on mouse studies, ≥50% LRRK2 knockdown required for phenotypic benefit |
| Safety margin | 10× the efficacious dose should show no severe adverse effects |
| Associated Diseases | Macaque, Rodents |
| Known Drugs/Compounds | nervous system, heart, gene silencing, gene editing, NDNF, Cas9 |
| KG Connections | 18 knowledge graph edges |
| Databases | GeneCardsUniProtNCBI GeneHPASTRING |