ニューヨーク・タイムズのインスタグラム(nytimes) - 2月13日 07時37分


Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in 1 errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent. With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure. In a half-dozen clinical trials planned or underway, researchers are testing strategies for correcting the problem at the genetic level. Among them is Brandon Williams, 21, who lives with his mother in Chicago. Because of his sickle-cell disease, he had suffered 4 strokes by age 18. The damage makes it hard for him to speak. His older sister died of the disease. Following an experimental gene therapy, his symptoms have vanished. It’s still early in the course of these experimental treatments, and it’s likely to be at least 3 years before one is approved. Although researchers hope the effects will last, they cannot be certain. @alyssaschukar took this photo of Brandon. Visit the link in our profile to see more.


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