Available Technologies
# of Displayed Technologies: 2 / 2
Applied Category Filter (Click To Remove): Gene Therapy
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
LNA Inhibitor Cocktail Therapy for Lupus
TS-037260 — A cocktail of locked nucleic acids inhibits the inflammatory response in a humanized murine model of lupus.
Systemic lupus erythematosus (SLE) is an orphan disease affecting approximately 5 in every 10,000 individuals. Despite its rare prevalence, this lifelong autoimmune disease has few effective targeted therapies. Current treatments are palliative using anti-inflammatory drugs or broad spectrum immun…
- College: College of Medicine (COM)
- Inventors: Jarjour, Wael; Valiente, Giancarlo; Young, Nicholas
- Licensing Officer: He, Panqing
