Advantages of RNA Trans-splicing for Editing Inherited & Acquired Mutations that Cause Disease

Time: 4:30 pm
day: Conference Day One


  • Describe the principles behind and approaches used for the creation of RNA transsplicing molecules (RTMs)
  • Review of cell culture and in vivo animal studies that demonstrate RTM editing of premessenger RNA and reversal of a wide variety of disease endpoints
  • How editing by RNA trans-splicing widens the therapeutic potential over other forms of RNA or DNA editing