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Rabbit Model for Duchenne Muscular Dystrophy
TS-058084 — A rabbit model has been created for Duchenne Muscular Dystrophy (DMD) that more closely resembles the effects experienced in humans while being more cost effective compared to larger animal models.
There has yet to be a DMD animal model that bridges the gap between cost effectiveness and scientific relevance. This new rabbit model does that. DMD is a lethal genetic disease characterized by progressive muscle weakness and muscle wasting. There are several animal models for DMD, includin…
  • College: College of Medicine (COM)
  • Inventors: Han, Renzhi
  • Licensing Officer: Schworer, Adam

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

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