Christine Bulawa

TL;DR: The molecular and structural basis of TTR tetramer stabilization by tafamidis is described, suggesting that binding stabilizes the weaker dimer-dimer interface against dissociation, the rate-limiting step of amyloidogenesis.

TL;DR: Tafamidis meglumine is a rationally designed, non-NSAID benzoxazole derivative that binds with high affinity and selectivity to TTR and kinetically stabilizes the tetramer, slowing monomer formation, misfolding, and amyloidogenesis.

TL;DR: Successful usage of RNA transcript therapy (RTT) is demonstrated as an exogenous human FXN supplementation strategy in vitro and in vivo, specifically to dorsal root ganglia (DRG), providing the first demonstration that RTT can be used for the delivery of therapeutic mRNA to DRG.

TL;DR: The structure reveals how FXN facilitates ISC production through stabilizing key loop conformations of NFS1 and ISCU at the protein–protein interfaces, and suggests how FRDA clinical mutations affect complex formation and FXN activation.

TL;DR: In this article, a method of treating a disorder characterized by impaired protein trafficking includes administering to a subject or contacting a cell with a compound of Formula I: [formula here] or pharmaceutically acceptable salts or derivatives thereof.