TS-005572 — This novel gene replacement strategy uses an adeno-associated viral vector to treat Cockayne syndrome type B (CSB). CSB is a rare neurodegenerative disorder caused by mutations in the ERCC6 or ERCC8 genes, both of which are responsible for DNA repair, causing tremors, ataxia, seizures, and cachectic dwarfism, significantly impacting their quality of life. An AAV-based gene replacement therapy will effectively combat this disorder by delivering a functional copy of the defective gene into target cells, thereby restoring normal gene expression.

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.