This page ranks genes associated with neurodegenerative diseases by various criteria including disease relevance, research activity, therapeutic target validation, and knowledge base coverage in NeuroWiki. [1]
| Rank | Gene | Key Functions | Research Activity |
|---|---|---|---|
| 1 | APP | Amyloid precursor protein, Aβ production | Very High |
| 2 | APOE | Lipid transport, amyloid clearance | Very High |
| 3 | PSEN1 | Gamma-secretase, Aβ generation | Very High |
| 4 | PSEN2 | Gamma-secretase, Aβ generation | High |
| 5 | MAPT | Tau protein, microtubule stability | Very High |
| 6 | TREM2 | Microglial activation, amyloid clearance | Very High |
| 7 | CLU | Clusterin, amyloid clearance | High |
| 8 | ABCA7 | Lipid transport, amyloid clearance | Moderate-High |
| 9 | SORL1 | APP trafficking, Aβ production | High |
| 10 | PLD3 | Lysosomal function, Aβ metabolism | Moderate |
| Rank | Gene | Key Functions | Research Activity |
|---|---|---|---|
| 1 | SNCA | Alpha-synuclein, Lewy body formation | Very High |
| 2 | LRRK2 | Leucine-rich repeat kinase 2 | Very High |
| 3 | GBA | Glucocerebrosidase, lysosomal function | Very High |
| 4 | PARK2 (Parkin) | Mitophagy, mitochondrial quality | High |
| 5 | PINK1 | Mitophagy, mitochondrial quality | High |
| 6 | DJ-1 | Oxidative stress response | Moderate-High |
| 7 | VPS35 | Endosomal trafficking | Moderate-High |
| 8 | DNAJC13 | Endosomal trafficking | Moderate |
| 9 | UCHL1 | Ubiquitin metabolism | Moderate |
| 10 | GIGYF2 | Insulin-like growth factor signaling | Moderate |
| Rank | Gene | Key Functions | Research Activity |
|---|---|---|---|
| 1 | C9orf72 | RNA metabolism, stress granules | Very High |
| 2 | SOD1 | Superoxide dismutase, oxidative stress | Very High |
| 3 | TARDBP (TDP-43) | RNA/DNA binding, splicing | Very High |
| 4 | FUS | RNA metabolism, stress granules | Very High |
| 5 | ANG | Angiogenin, stress response | High |
| 6 | UBQLN2 | Protein degradation | Moderate-High |
| 7 | OPTN | Autophagy, mitophagy | Moderate-High |
| 8 | TBK1 | Autophagy, inflammation | Moderate-High |
| 9 | VCP | Protein degradation, autophagy | Moderate |
| 10 | HNRNPA1 | RNA metabolism | Moderate |
Genes with strong evidence for therapeutic targeting:
| Rank | Gene | Target Approach | Validation Level |
|---|---|---|---|
| 1 | APP/Aβ | Immunotherapy, BACE inhibitors | Clinical |
| 2 | MAPT/Tau | Immunotherapy, kinase inhibitors | Clinical |
| 3 | SNCA | Immunotherapy, gene therapy | Clinical |
| 4 | TREM2 | Agonist antibodies | Preclinical |
| 5 | LRRK2 | Kinase inhibitors | Clinical |
| 6 | GBA | Enzyme enhancement | Clinical |
| 7 | SOD1 | Gene therapy, ASO | Clinical |
| 8 | C9orf72 | Gene therapy, ASO | Preclinical |
These ranked items have limited content and would benefit from expansion:
APP and Alzheimer's disease. Nat Rev Neurosci. 2013. ↩︎