基因编辑技术为唐氏综合征治疗提供新思路

Core Insights - A research team from Mie University and other institutions in Japan has successfully utilized gene editing technology to remove the extra 21st chromosome from cells of patients with Down syndrome, confirming the results in a publication in the Proceedings of the National Academy of Sciences [1][2] Group 1: Research Methodology - The team extracted fibroblasts from the skin of Down syndrome patients to cultivate induced pluripotent stem cells (iPS cells) [2] - They developed three types of iPS cells, each with one of the three 21st chromosomes deleted, and constructed a CRISPR/Cas9 system to target and cut the extra chromosome at multiple sites [2] - The CRISPR/Cas9 system achieved a removal accuracy of up to 37.5% for the target 21st chromosome [2] Group 2: Findings and Implications - Analysis of the modified iPS cells showed that their characteristics, including gene expression patterns, cell proliferation rates, and reactive oxygen handling, had returned to normal [2] - The CRISPR/Cas9 system was also confirmed to be effective in removing chromosomes from differentiated cells, such as fibroblasts and non-dividing cells [2] Group 3: Future Directions - The technology is currently in the concept validation stage through in vitro cell experiments and has some limitations [2] - Future research will focus on developing safer chromosome removal techniques that do not rely on cutting [2]