精准基因治疗

Search documents
AI设计出特定类型细胞“基因开关”
Ke Ji Ri Bao· 2025-05-11 01:10
Group 1 - A new study published in the journal "Cell" highlights the use of generative artificial intelligence (AI) to design synthetic DNA sequences that act as "gene switches" to control gene expression in specific mammalian cells, marking a milestone in synthetic biology [1][2] - The AI model was trained to predict suitable combinations of DNA letters (A, T, C, G) to create DNA fragments that do not exist in nature, allowing for tailored gene expression patterns in specific cell types, such as enabling stem cells to develop into red blood cells instead of platelets [1][2] - The research team conducted over 64,000 experiments over five years to build the largest synthetic enhancer database for blood cells, testing 38 different transcription factor binding site arrangements and tracking enhancer activity across seven stages of blood cell development [2] Group 2 - The ability to create artificial enhancers that function as precise "on/off" switches in specific cells significantly reduces side effects on healthy cells, which is crucial for the development of next-generation gene therapies [2] - The study revealed interesting phenomena, such as some enhancer combinations exhibiting "negative synergistic effects," where two factors that individually promote gene expression can inhibit activity when combined, providing new insights into gene regulation [2][3] - This advancement opens new avenues for personalized medicine and precision gene therapy in the future [3]