What Happened
Almost as soon as researchers started exploring the capabilities of the CRISPR/Cas9 system, they recognized its potential use in targeted gene editing.
Why It Matters
But the intervening decades have seen slow progress as people worked to determine how to do so in a way that would be safe for use in humans.
Key Details
- It was only a little over two years ago, decades after CRISPR's discovery, that the FDA approved the first CRISPR-based therapy, for sickle-cell anemia.
- Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene editing system that produces more focused changes and fewer mistakes.
- And they've used it to produce a therapy that addresses a disease that's closely related to sickle-cell anemia: β-Thalassaemia.
- Gene editing and its limits The CRISPR/Cas-9 system provides bacteria with a form of immunity.
Background Context
Almost as soon as researchers started exploring the capabilities of the CRISPR/Cas9 system, they recognized its potential use in targeted gene editing. But the intervening decades have seen slow progress as people worked to determine how to do so in a way that would be safe for use in humans. It was only a little over two years ago, decades after CRISPR's discovery, that the FDA approved the first CRISPR-based therapy, for sickle-cell anemia. Now, following up on that success, a large Chinese collaboration has followed up with a description of an improved gene editing system that produces more focused changes and fewer mistakes. And they've used it to produce a therapy that addresses a disea
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Source: Ars Technica – All content – Original Link
Source: Ars Technica – All content
