AIMers: Correcting Driver Mutations for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) and Beyond

Time: 11:30 am
day: Day Two


  • Understand how our RNA editing platform allows for correction of driver mutations of AATD
  • Developing RNA Editing AIMers to correct homozygous “ZZ” mutations back to “MZ” heterozygous state
  • AIMers lead to durable and functional expression of M-AAT protein, establishing an RNA editing platform to target liver and potentially other organs