# of Displayed Technologies: 3 / 3

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Mini-PCDH15 for treatment of deafness
TS-050433 — A gene therapy solution for Usher Syndrome Type 1F.
Mutations in PCDH15 cause Usher 1F, a recessive syndrome characterized by profound congenital deafness and absence of vestibular function, and progressive blindness beginning in the second decade. Because patients who lack hearing and balance rely on vision for communication and mobility, the late…
  • College: College of Arts & Sciences
  • Inventors: Sotomayor, Marcos
  • Licensing Officer: Ezzell, Janel

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-targeting activity, and an intein split for packaging into an adeno-associated virus (AAV), resulting in gene mutation correction.
There are thousands of monogenic diseases such as Duchenne muscular dystrophy (DMD) lacking effective therapeutic treatments. DMD is caused by genetic mutations in the DMD gene, which encodes the dystrophin protein. The mutations including missense point mutations, nonsense point mutations, deleti…
  • College: College of Medicine (COM)
  • Inventors: Han, Renzhi; Gao, Yandi; Xu, Li
  • Licensing Officer: Davis, Stewart

Gene delivery to adipose tissue using novel recombinant adeno-associated viral (AAV) vectors
TS-037310 — An engineered recombinant adeno-associated virus capable of targeting visceral fat tissue for the purposes of genetic manipulation.
Recombinant adeno-associated viruses (rAAVs) are effective tools for delivering gene therapy treatment and for manipulation of acquired diseases. However, there is no effective method to target adipose tissue and transduction efficiency is low in naturally occurring AAV serotypes. In addition, cur…
  • College: College of Medicine (COM)
  • Inventors: Cao, Lei; Huang, Wei
  • Licensing Officer: Ezzell, Janel

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