登上Cell子刊封面:复旦大学开发出最强Fanzor基因编辑系统,单个AAV递送,实现高效体内基因编辑
生物世界·2025-11-30 08:00

Core Viewpoint - The article discusses the development and potential of the Fanzor system, a compact RNA-guided DNA nuclease, for precise genome editing in eukaryotic cells, highlighting its advantages over traditional Cas9 systems and its application in disease treatment [3][9]. Group 1: Fanzor System Overview - Fanzor is a newly discovered CRISPR-like system that allows for precise genome editing in eukaryotic organisms, with a compact size of 400-700 amino acids, making it suitable for delivery via a single AAV vector [3][9]. - The traditional AAV delivery system has a maximum packaging capacity of 4.7 kb, which is insufficient for larger Cas proteins like SpCas9 and AsCas12a, necessitating the use of dual AAV vectors [3]. Group 2: Research Developments - A study published by a team from Fudan University introduced an engineered hypercompact Fanzor-ωRNA system, which demonstrated enhanced genome editing activity, achieving efficiency improvements of 6-129 times compared to previous versions [4][11]. - The engineered SpuFz1 V4 system, derived from soil fungus, showed strong genome editing capabilities in human cells, marking it as the most active member of the Fanzor family [9][11]. Group 3: Applications and Potential - The compact structure of SpuFz1 V4 allows for effective in vivo delivery using a single AAV, enabling robust genome editing in animal models, such as efficient editing of the Nfkb1 gene in mouse retinas [10][11]. - The research indicates that the Fanzor platform can be developed into base editors, facilitating efficient single-base editing, which has significant implications for genetic research and therapeutic applications [5][9].