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Last summer, doctors in Tennessee injected Victoria Gray—a 34-year-old sickle cell disease patient —with billions of her own stem cells that scientists in Massachusetts had reprogrammed with Crispr to produce healthy blood cells. The hours-long infusion made her the first American with a heritable disease to be treated with the experimental gene-editing technology. And it appears to be working.
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In December, a team that includes researchers from CRISPR Therapeutics and Vertex Pharmaceuticals published promising results from a clinical trial, which is also treating patients in Germany who suffer from a related disease called ß-thalassaemia. In both groups of patients, the treatment seems to be safe, and it so far has eliminated the need for regular blood transfusions. It’s still too soon to say how long the effects will last but the consequences could be huge. Sickle cell disease and ß-thalassaemia are among the most common genetic disorders caused by mutations to a single gene, affecting millions of people worldwide.
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For thousands of years, humans have been modifying the DNA of our closest furry and feathered friends by breeding animals to produce the most desirable traits. With Crispr, one no longer has to wait generations to make significant genetic changes. This year, researchers 'created' pandemic-proof pigs , whose cells have been edited to remove the molecular lock-and-key mechanism that a variety of respiratory viruses use to infect them, and chickens Crispr’d to make them impervious to a common bird disease caused by the avian leukosis virus.
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It would be beneficial to develop commercial diagnostics without the need for expensive lab instruments by using Crispr’s programmable gene-seeking capabilities to pick up bits of foreign genetic material—from a virus, bacteria, or fungus—circulating in a sick person’s bodily fluids, and deliver those results via something that looks like a pregnancy test. Tests made with disposable paper strips are cheap and can go into the field or into people’s homes, greatly expanding their reach. This summer, the FDA authorized two Crispr-based tests, both for detecting SARS-CoV-2. Boston-based (Sherlock Biosciences, May, and the Bay Area’s Mammoth Biosciences, August). It marked the first time the FDA has allowed a Crispr-based diagnostic tool to be used on patients. The tests still need to be analyzed in a lab, but they are faster than the standard method for detecting SARS-CoV-2, called PCR.
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Unlike chromosomes, which you inherit from both parents, mitochondrial DNA comes only from your maternal side. Mutations can cripple the cell’s ability to generate energy and lead to debilitating, often fatal conditions that affect about one in 6,500 people worldwide. Until now, scientists prevent mitochondrial disease by swapping out one egg’s mitochondria for another, a procedure commonly known as three-person IVF . This summer, scientists in Seattle and Boston published a study showing they had discovered a way to harness a strange enzyme found in biofilm-forming bacteria to make precise changes to mitochondrial DNA. The new system has not yet been tested in humans but the discovery opens up another promising avenue.
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The 2020 Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer Doudna (first women to win prizes in science together) for Crispr genome editing. It was both a stunning choice (as a DNA-altering tool, Crispr has only been around for 8 years) and a completely expected one. Crispr has completely revolutionized biological research since its arrival in 2012 ; scientists have since published more than 300,000 studies using the tool to manipulate the genomes of organisms across every kingdom. It’s cheap, fast, and easy enough for almost anyone to use.
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