Can gene therapy really cure disease?
Quick answer: Yes, in specific cases—approved gene therapies now treat spinal muscular atrophy, certain inherited blindness, hemophilia, and sickle cell disease. They are…
Yes, in specific cases—approved gene therapies now treat spinal muscular atrophy, certain inherited blindness, hemophilia, and sickle cell disease. They are one-time treatments with extraordinary prices, and research is expanding to more conditions.
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