Targeted Drug Delivert Proposal for Ataxia-Telangiectasia (A-T)

Developed a groundbreaking drug delivery approach that combines blood–brain-barrier–penetrating lipid nanoparticles (LNPs) with antisense oligonucleotides (ASOs) to correct gene splicing errors in ataxia-telangiectasia, presenting one of the first therapeutic strategies with the potential to move beyond symptom management toward a curative treatment for this rare and underserved neurodegenerative disorder.