One in 5,500 newborns are affected by Tuberous Sclerosis Complex (TSC), a multi-organ developmental disorder caused by mutations in either the TSC1 or TSC2 gene. Nationwide Children's Hospital principal investigator, Mark Hester, developed a novel gene therapy strategy that uses an adeno-associated viral (AAV) vector to treat TSC2. Due to the large gene size of TSC2 and limited packaging space of AAV vectors, he designed micro-Tuberin candidates with similar structure and functional capacity as full-length Tuberin candidates. Final constructs were then packaged into AAV9 viral vectors to test their ability to suppress mTORC1 signaling.