International Journal of Molecular Zoology 2024, Vol.14, No.1, 9-17 http://animalscipublisher.com/index.php/ijmz 12 techniques to repair or replace critical genes, restoring visual function in mice. This provides a robust foundation for the development of potential genetic therapeutic approaches. In mouse models, researchers can conduct drug screening experiments to identify potential drug treatment options. These trials help evaluate the efficacy of different drugs in alleviating blindness symptoms, providing crucial information for clinical treatments. Optogenetics, a method based on light-sensitive proteins, is used to control neuronal activity. Mouse models play a vital role in optogenetic research, aiding in the understanding of the visual system's functionality and the development of new therapeutic approaches. Blindness involves not only damage to retinal cells but also requires neuroprotective strategies. Mouse models are employed to study the effectiveness of neuroprotective methods to slow down or halt the progression of blindness. In conclusion, mouse models play a crucial role in hereditary blindness research, offering researchers opportunities to gain in-depth insights into blindness mechanisms and test potential therapeutic methods. Combined with advanced tools such as gene-editing technologies, mouse models hold the promise of bringing new breakthroughs in the treatment of hereditary blindness. 3 Development and Application of Gene Editing Technologies 3.1 Overview of CRISPR/Cas9 gene editing technology and its principles Since its inception, the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/Cas9) gene editing technology has garnered widespread attention and sparked a revolution in the scientific community. This technology allows scientists to precisely modify gene sequences, providing a powerful tool for researching genetic diseases and exploring gene therapy. The principle of CRISPR/Cas9 is based on a bacterial defense mechanism found in the natural immune system, which can identify and cut the genome of invading viruses. CRISPR represents a series of short DNA sequences, while Cas9 is an enzyme capable of cutting DNA. During the gene editing process, researchers design a CRISPR guide RNA that can recognize specific gene sequences. Once the CRISPR guide RNA matches the target gene, the Cas9 enzyme is activated to cut the target DNA. Cells then attempt to repair the cut DNA, usually through non-homologous end joining (NHEJ) or homology-directed repair (HDR). These repair processes may result in gene mutations, achieving the goal of gene editing (Figure 3). Figure 3 Gene editing process (Source: Wikipedia)
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