ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and Via LNPs in Ex Vivo Cortical Neurons.

Bethany A. Adair,Andrea J. Korecki,Diana Djaksigulova,Pamela K. Wagner, Nina Y. Chiu,Siu Ling Lam,Tess C. Lengyell,Blair R. Leavitt,Elizabeth M. Simpson

Ophthalmology and Therapy（2023）

引用 1|浏览27

关键词

Adenine base editor,Aniridia,CRISPR gene therapy,Humanization,Lipid nanoparticles,Mouse embryonic stem cells,Paired box 6 (PAX6),Primary cortical neurons

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