Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus - Cui, Tongtong; Cai, Bingyu; Tian, Yao; Liu, Xin; Liang, Chen; Gao, Qingqin; Li, Bojin; Ding, Yali; Li, Rongqi; Zhou, Qi; Li, Wei; Teng, Fei (Advanced science, 2024)
Keywords: AAV delivery; Amino acids; Animals; CRISPR; CRISPR-Cas Systems - genetics; CRISPR‐Cas12f1; Dependovirus - genetics; Disease Models, Animal; Efficiency; gene editing; Gene Editing - methods; Gene therapy; Genetic disorders; Genetic Therapy - methods; Genetic Vectors - genetics; Genomes; Humans; Mice; Mutation; Photoreceptors; Proteins; retinitis pigmentosa; Retinitis Pigmentosa - genetics; Retinitis Pigmentosa - therapy; Vectors (Biology); retinitis pigmentosa; gene therapy; AAV delivery; gene editing; CRISPR‐Cas12f1