| #1 |
Transcriptional Autophagy-Lysosome Coupling |
FOXO1 |
0.669 |
$0.67 |
| FOXO1 coordinates autophagy gene expression with lysosomal biogenesis through dual regulation of ATG genes and TFEB nuclear translocation. Selective FOXO1 activation could synchronize the entire autop... |
| #2 |
Autophagosome Maturation Checkpoint Control |
STX17 |
0.616 |
$0.62 |
| STX17 controls autophagosome-lysosome fusion through SNARE complex formation. Developing STX17 activity enhancers could overcome fusion bottlenecks that accumulate immature autophagosomes in NDDs.... |
| #3 |
Lysosomal Enzyme Trafficking Correction |
IGF2R |
0.605 |
$0.60 |
| M6PR trafficking defects cause lysosomal enzyme mislocalization. Pharmacological chaperones that stabilize M6PR-enzyme complexes could restore proper enzyme delivery to lysosomes.... |
| #4 |
Lysosomal Calcium Channel Modulation Therapy |
MCOLN1 |
0.595 |
$0.59 |
| TRPML1 calcium channels regulate lysosomal biogenesis and autophagosome-lysosome fusion. Pharmacological enhancement of TRPML1 activity could restore lysosomal function across multiple NDDs.... |
| #5 |
Lysosomal Membrane Repair Enhancement |
CHMP2B |
0.553 |
$0.55 |
| ESCRT-III machinery repairs damaged lysosomal membranes to prevent cytotoxic content leakage. Upregulating CHMP2B and coordinating ESCRT components could maintain lysosomal integrity under proteotoxic... |
| #6 |
Mitochondrial-Lysosomal Contact Site Engineering |
RAB7A |
0.528 |
$0.53 |
| RAB7 and PRKN coordinate mitochondrial-lysosomal membrane contact sites essential for mitophagy. Engineering enhanced MLCS formation through stabilized RAB7-PRKN interactions could bypass upstream aut... |
| #7 |
Lysosomal Positioning Dynamics Modulation |
LAMP1 |
0.498 |
$0.50 |
| LAMP1-dynein motor complex positioning determines lysosomal distribution and fusion efficiency. Enhancing LAMP1-mediated lysosomal retrograde transport could improve autophagosome-lysosome encounters ... |