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Dr. Dominic Ng

Dr. Dominic Ng
@DrDominicNg

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While everyone was obsessing over CRISPR, a small team just quietly published a paper in Science solving genetic medicine's biggest problem. They created a system that can fix thousands of different mutations at once. Here's how they did it 🧵

Current gene editing 101: You inherit a disease-causing mutation → CRISPR-Cas9 targets that exact DNA sequence → cleaves both strands → cell repairs it with correct template. Already curing sickle cell. Already reversing genetic blindness. Already changing medicine.
But CRISPR has a massive blind spot: it needs to know EXACTLY which mutation to fix. Problem is, most genetic diseases aren't that simple. Cystic fibrosis? Over 2,000 different mutations. Same disease, different typos in every patient. Now what?
That's where this breakthrough comes in. Researchers turbocharged a bacterial system (CAST) that doesn't edit mutations at all—it delivers a fresh, working gene copy to a safe spot in your DNA. The broken version? Still there, just overruled. 200x more efficient than before.
The results are stunning: They inserted full-length genes into "safe harbour" sites (think: DNA parking spots that won't cause problems). Engineered CAR-T cells for cancer. Delivered packages over 10,000 DNA letters long. Worked across multiple cell types. Zero genomic scars.
Bottom line: Those 2,000 cystic fibrosis mutations that make CRISPR impractical? Irrelevant now. This delivers a working gene that drowns out every broken version. One shot treats them all. We're not editing diseases anymore—we're overwriting them.
Dr. Dominic Ng

Dr. Dominic Ng

@DrDominicNg
Medical Doctor | PhD candidate decoding the brain | Academic writer distilling complex neuroscience into concise, peer-reviewed insights
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