Scientists have improved the vision of a small number of patients suffering from a rare and incurable eye disease by replacing a defective gene with a healthy one-a boost for a technique known as gene therapy.
通过将有缺陷的基因替换成健康的基因,科学家已经使少数患有一种罕见、难愈眼病的病人改善了视力。这对一项被称为基因疗法的技术是个推动。
The patients have choroideremia, a degenerative disease caused by defects in a single gene that leads to blindness and affects 1 in 50,000 people. In an early-stage trial published Wednesday in the Lancet, the researchers used a deactivated virus to safely ferry billions of healthy, lab-made versions of the gene into the retina. That appeared to restore the function of light-sensitive cells, which the disease impairs.
上述病人患有脉络膜缺失症。这是一种由单一基因缺陷造成的退化性疾病,会导致失明,每50,000人就有1例。医学期刊《柳叶刀》(Lancet)周三刊登了初步试验的结果。在试验中,研究人员使用了一种失去活性的病毒来携带实验室制作的健康基因,将数以十亿计的这类基因安全地注入视网膜。这在表观上恢复了疾病损害的感光细胞的功能。
'We were surprised by the magnitude of vision improvement' in the patients, said Robert MacLaren, a professor of ophthalmology at the University of Oxford in England and leader of the clinical trial.
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