ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons.
Bethany A AdairAndrea J KoreckiDiana DjaksigulovaPamela K WagnerNina Y ChiuSiu Ling LamTess C LengyellBlair R LeavittElizabeth M SimpsonPublished in: Ophthalmology and therapy (2023)
We demonstrated the usefulness of the CHuMMMs approach, and showed the first genomic editing by ABE8e encapsulated as an LNP-RNP. Furthermore, we laid the foundation for translation of the proposed CRISPR therapy to preclinical mouse studies and eventually patients with aniridia.