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…
