TS-004704 — This IP automates the miniaturization of large gene targets through a structure-based protein engineering AI algorithm.

TS-004616 — A method for targeted delivery and constitutive expression of large genes using adeno-associated viral (AAVs) vectors that contain ubiquitous mini-promoters. Incorporation of the compact mini-promoters will increase the cargo space of the AAVs such that larger genes can be packaged into the vectors.

TS-002646 — 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.