Diseases & Conditions

Whatever happened to CRISPR?

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No disease has yet been cured by CRISPR, but I believe cures are coming. The question is how long it will take. The diseases that should be easiest to cure are those caused by a defect in the structure of a single gene. There aren't a lot of such diseases, but there are some. An example is sickle cell disease. Children born with the gene defect that causes this disease are cursed by bouts of terrible pain, severe anemia that saps their strength and energy, organ damage, even death. And it's all caused by a tiny defect. How tiny? Imagine all of the DNA in one of your cells as an amplified strand that stretches from Boston to San Francisco. The defect that causes the disease is just the size of a suitcase. All the rest of the DNA, from Boston to San Francisco, is fine. CRISPR would enable scientists to precisely fix just that tiny defective part of the gene, leaving all the rest of the DNA unchanged.

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