首页|AAV-mediated gene therapies by miniature gene editing tools
AAV-mediated gene therapies by miniature gene editing tools
扫码查看
点击上方二维码区域，可以放大扫码查看
原文链接
万方数据
维普
The advent of CRISPR-Cas has revolutionized precise gene editing.While pioneering CRISPR nucleases like Cas9 and Cas12 generate targeted DNA double-strand breaks(DSB)for knockout or homology-directed repair,next generation CRISPR technologies enable gene editing without DNA DSB.Base editors directly convert bases,prime editors make diverse alterations,and dead Cas-regulator fusions allow nuanced control of gene expression,avoiding potentially risks like translocations.Meanwhile,the discovery of diminutive Cas12 orthologs and Obligate Mobile Element-Guided Activity(OMEGA)nucleases has overcome cargo limitations of adeno-associated viral vectors,ex-panding prospects for in vivo therapeutic delivery.Here,we review the ever-evolving landscape of cutting-edge gene editing tools,focusing on miniature Cas12 orthologs and OMEGA effectors amenable to single AAV packaging.We also summarize CRISPR therapies delivered using AAV vectors,discuss challenges such as efficiency and specificity,and look to the future of this transformative field of in vivo gene editing enabled by AAV vectors delivery.
gene therapyadeno-associated viruses(AAVs)genome editingminiature CRISPR-CasObligate Mobile Element-Guided Activity(OMEGA)
Xiangfeng Kong、Tong Li、Hui Yang
展开 >
Shanghai Institute of Materia Medica,Chinese Academy of Sciences,Shanghai 201203,China
Institute of Neuroscience,Center for Excellence in Brain Science and Intelligence Technology,Chinese Academy of Sciences,Shanghai 200031,China
万方数据
维普
