Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus

• Published in: Advanced science Vol. 11; no. 19; pp. e2308095 - n/a
• Main Authors: Cui, Tongtong, Cai, Bingyu, Tian, Yao, Liu, Xin, Liang, Chen, Gao, Qingqin, Li, Bojin, Ding, Yali, Li, Rongqi, Zhou, Qi, Li, Wei, Teng, Fei
• Format: Journal Article
• Language: English
• Published: Germany John Wiley & Sons, Inc 01.05.2024; John Wiley and Sons Inc; Wiley
• Subjects: 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

CRISPR‐based gene therapies are making remarkable strides toward the clinic. But the large size of most widely used Cas endonucleases including Cas9 and Cas12a restricts their efficient delivery by the adeno‐associated virus (AAV) for in vivo gene editing. Being exceptionally small, the recently eng...