A Luciferase Reporter Mouse Model to Optimize in Vivo Gene Editing Validated by Lipid Nanoparticle Delivery of Adenine Base Editors.
Molecular Therapy（2023）
关键词
gene therapy,gene editing,base editing,CRISPR-Cas9,in vivo gene editing,nonviral delivery,lipid nanoparticles,mouse models,CRISPR,Cas9
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