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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-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

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