These diseases are a good comparison point because they’re relatively easy to treat…Researchers have thrown a number of gene-editing techniques at these diseases…[b]ut if you ask experts in the field, the smart money’s on Crispr.
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With Crispr, since cells are being brought outside the body and treated in the lab, it’s easier to ensure that it’s the actual stem cells that are being edited. And if a Crispr team can get a higher fraction of edited stem cells to persist in bone marrow, a one-time treatment could permanently alleviate a blood disorder.
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A huge advantage of the technique is what led to its mass adoption in the lab—the gene-editing systems are simple and easy to make. PNAs, on the other hand, involve complex chemistry reminiscent of zinc finger nucleases (ZFNs)….
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