Fig. 3
From: Nuclear-import receptors as gatekeepers of pathological phase transitions in ALS/FTD
Designing and engineering NIR-based drug candidates with optimized activity and suitable properties for delivery. Starting from naturally occurring, wild-type NIRs, specific cargo binding, improved chaperoning activity and increased NPC transition rates can be achieved by generating variants with altered cargo-binding affinities and amino acid substitutions at the NIR surface. Smaller, active NIR fragments will facilitate NIR gene delivery via AAVs and could exhibit lower immunogenicity. PDB-ID: 2H4M