Diverse perspectives on science and medicine
By the time that the FDA’s advisory committee gave a near-instantaneous and unanimous thumbs-up for gene therapy to treat a form of hereditary blindness on October 12, tears were freely flowing. Fittingly, it was World Sight
On a spectacular September Sunday in 2008, 8-year-old Corey Haas, a cane in one hand and his mother’s hand in the other, stepped tentatively on the pathway leading into the Philadelphia zoo. Hearing kids yelling
Three years ago, at a fundraiser near Philadelphia for the Curing Retinal Blindness Foundation, I stood, dumbstruck, as young teen Christian Guardino took the stage and belted out Don’t Stop Believing. Christian had recently undergone
Here’s an update on some of the rare disease families DNA Science has covered as they travel the long and winding road from diagnosis to gene therapy. The Challenge The rarity of many single-gene diseases
The news this week presented at a major vision conference and published in The New England Journal of Medicine about gene therapy to treat childhood blindness paints an unnecessarily grim picture. Because I wrote a book
I am astonished, once again, by the complexity and unpredictability of science. Last week, a paper in the Proceedings of the National Academy of Science (PNAS) reported that gene therapy to treat a form of