Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia

por Mirjalili Mohanna, Seyedeh Zeinab, Hickmott, Jack W., Lam, Siu Ling, Chiu, Nina Y., Lengyell, Tess C., Tam, Beatrice M., Moritz, Orson L., Simpson, Elizabeth M.
Publicado 2020

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

por Adair, Bethany A., Korecki, Andrea J., Djaksigulova, Diana, Wagner, Pamela K., Chiu, Nina Y., Lam, Siu Ling, Lengyell, Tess C., Leavitt, Blair R., Simpson, Elizabeth M.
Publicado 2023