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下一代遗传医学平台:该新型工具具有治疗多种遗传疾病的潜力
科学家发现了一种基因编辑技术,能够有效和准确地纠正某些疾病的遗传缺陷,将新工具定位为下一代基因疗法的基础。该编辑平台可用于治疗遗传性和后天性疾病。概念证明研究聚焦了一种有前途的新基因编辑平台,该平台最终可用于治疗镰状细胞病、血友病和其他遗传疾病等疾病。
https://www.sciencedaily.com/releases/2018/07/180716151519.htm
Laura J. Smith, Jason Wright, Gabriella Clark, Taihra Ul-Hasan, Xiangyang Jin, Abigail Fong, Manasa Chandra, Thia St Martin, Hillard Rubin, David Knowlton, Jeff L. Ellsworth, Yuman Fong, Kamehameha K. Wong, Saswati Chatterjee. Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing. Proceedings of the National Academy of Sciences, 2018; 201802343 DOI: 10.1073/pnas.1802343115
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肠道细菌的单一遗传改变改变宿主的新陈代谢
科学家们发现,在一种特殊的肠道细菌中删除一个基因会导致小鼠体内新陈代谢的改变和体重的降低。
https://www.sciencedaily.com/releases/2018/07/180717135704.htm
Lina Yao, Sarah Craven Seaton, Sula Ndousse-Fetter, Arijit A Adhikari, Nicholas DiBenedetto, Amir I Mina, Alexander S Banks, Lynn Bry, A Sloan Devlin. A selective gut bacterial bile salt hydrolase alters host metabolism. eLife, 2018; 7 DOI: 10.7554/eLife.37182
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