CRISPR/Cas9 is a gene editing technology that has emerged in recent years, allowing precise modifications of specific DNA sequences. Due to its simplicity and high efficiency, it quickly gained widespread attention in the scientific community upon its introduction. As it has evolved, various novel CRISPR-based gene editing techniques have been continuously developed, including gene activation/inhibition of endogenous genes, genetic labeling, epigenetic modifications, single-base editing, and more. In addition to DNA-targeting gene editing systems, RNA-targeting CRISPR systems have also been recently developed. These systems enable the regulation of targeted gene expression without disrupting DNA sequences, showing great promise in the field of disease therapy. 

Our research group primarily focuses on the following two directions: 

1. Development and Optimization of Gene Editing Tools: 

Despite the widespread use of CRISPR-based gene editing technology across various fields of life science research, there are still several limitations that require further improvement. For example, gene editing systems are generally large, which significantly hampers their delivery in vivo. Moreover, existing gene editing techniques often exhibit potential off-target effects or insufficient editing efficiency. In our future work, our research group will utilize bioinformatics to discover novel CRISPR-based gene editing systems from nature and modify existing gene editing tools. We aim to optimize gene editing technology to achieve safer and more efficient tools, laying the foundation for future clinical applications. 

2. Application of Gene Editing Technology in Treating Major Neurological Disorders: 

The ultimate goal of our research group is to translate these emerging gene editing techniques into methods for improving human health. Neurological disorders constitute a significant threat to the quality of life and lifespan of individuals, including common conditions such as Alzheimer's disease (AD) and Parkinson's disease (PD). Currently, there are limited or no curative methods for many severe neurological disorders in clinical practice. Our previous work demonstrated that the application of gene editing technology can directly convert glial cells into specific types of neurons in vivo, alleviating permanent visual impairment and symptoms of Parkinson's disease. In our future work, we will first apply gene editing technology to treat major neurological disorders in mouse disease models. After successful validation in mouse models, we will extend these approaches to non-human primate disease models.