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An improved base editor (iABE-NG) for correcting genetic diseases such as Duchenne muscular dystrophy
TS-049505 — An engineered base editor (iABE-NG) that uses an SpCas9 variant, a shortened adenine deaminase domain with eliminated RNA off-target activity, and an intein split for packaging into an adeno-associated virus (AAV), resulting in gene mutation correction.
There are thousands of monogenic diseases that are lacking effective therapeutic treatments. The main bottleneck in developing effective treatment is the ability to effectively edit gene while controlling for the myriad of off-target effects that come with targeting the genome. Duchenne Muscular D…
  • College: College of Medicine (COM)
  • Inventors: Han, Renzhi; Gao, Yandi; Xu, Li
  • Licensing Officer: Schworer, Adam

Sustained Release Microparticles for the Treatment of Ocular Diseases
TS-038312 — A novel, intravitreal microparticle injection capable of controlled-release of protein therapies (including anti-VEGF) for tunable periods of 6-12 months after injection for the treatment of age-related macular degeneration.
Age-related macular degeneration (AMD) is the leading cause of blindness in individuals 65 and older and is the third leading cause of blindness worldwide. Wet AMD is characterized by abnormal growth of leaky blood vessels leading to damage to the macula of the retina resulting in vision loss and …
  • College: College of Engineering (COE)
  • Inventors: Jiang, Pengfei; Ohr, Matthew; Reilly, Katelyn
  • Licensing Officer: Schworer, Adam

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