Base editors: development and applications in biomedicine / 医学前沿

Base editor (BE) is a gene-editing tool developed by combining the CRISPR/Cas system with an individual deaminase, enabling precise single-base substitution in DNA or RNA without generating a DNA double-strand break (DSB) or requiring donor DNA templates in living cells. Base editors offer more precise and secure genome-editing effects than other conventional artificial nuclease systems, such as CRISPR/Cas9, as the DSB induced by Cas9 will cause severe damage to the genome. Thus, base editors have important applications in the field of biomedicine, including gene function investigation, directed protein evolution, genetic lineage tracing, disease modeling, and gene therapy. Since the development of the two main base editors, cytosine base editors (CBEs) and adenine base editors (ABEs), scientists have developed more than 100 optimized base editors with improved editing efficiency, precision, specificity, targeting scope, and capacity to be delivered in vivo, greatly enhancing their application potential in biomedicine. Here, we review the recent development of base editors, summarize their applications in the biomedical field, and discuss future perspectives and challenges for therapeutic applications.

Establishment and application of genetically modified pig tool models / 中国实验动物学报

Genetically modified tool animal models are the animal models,which are generated by modifying a defined gene and can be used as a tool to help realize other objective.Genetically modified large animals have wide applications in agriculture and biomedicine.Tool animal models play important role in biological research and development of new drugs in biomedicine,especially,have made tremendous contribution in revealing gene function and pathway of signal transduction.Pigs are not only an economically important agriculture animals,but also an ideal animal model in biomedicine due to its close similarity to human in physiology,as well as organ structure and size.Thanks to the breakthrough of newly emerged gene editing technology,striking progress has made in establishment of genetically modified tool pig models which include the ones used for monitoring pluripotency of cells,tracing cell lineages,replacing genes mediated by Cre recombinase,mimicking immunodeficiency,as well as gene editing in vivo.These tool models have been widely applied in biological research.Here,we will review the progress in generation of genetically modified tool pig models and their applications.