VOA常速英语:新研究：镰刀形贫血症或可治愈

Twenty-nine-year-old Lynndrick Holmes of Mobile Alabama says sickle cell anemia made him feel like his body was suffocating. He couldn’t do much. So I wanted to play football. I wanted to play basketball. I wanted to do karate and Taekwondo. But I couldn’t. So you just in this. You just vicariously living through your peers. Most of my life, I just vicariously live through the people that were around me and I watched them live lives that I couldn’t. But no longer. Holmes was one of nine patients in a small clinical trial of a new treatment for sickle cell at the National Institutes of Health. Healthy red blood cells around flat disks that flow easily through the body’s blood vessels. In sickle cell, disease protein crystals formed inside the cells, changing them to a crescent-shape, clogging vessels and causing pain and organ damage. The disease is caused by a defective gene.

Rather than editing the gene out, Dr. John Tisdale says his team added a copy of the normal correct gene which produced healthy blood cells. We’re simply adding the gene. We leave the defective gene intact. So this is what’s called gene addition therapy. Within two or three months, all nine patients showed signs of remission, and were starting to discover normal health for the first time in their lives. I’m not the same Lynndrick you know. So I’m like, yo, that happened. I was a part of it and I don’t have sickle cell anymore. NIH director Francis Collins called that spectacular. These patients who had sickle cell disease, now when you look at their blood counts and their blood smears, it looks like they don’t have it anymore. Now it’s early days. We’ve only followed these patients for a year at the most. But it looks like it would be fair to say this could be a cure.
Not overnight, he adds. And with the technology and cost involved in this trial, it’s unlikely to help sickle cell patients in poor countries. But the approach of introducing a corrected gene holds promise for all nations as researchers continue to refine and improve the therapy.
Faith Lapidus VOA News.