CRISPR Used to Treat Rare Genetic Disease for First Time

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In March of last year doctors in Oregon used CRISPR inside a person’s body for the first time. Now, another team of doctors, this time in England, has used the revolutionary gene editing tool in vivo once again. This time for a trial attempting to treat people with a rare genetic disorder that causes the buildup of a harmful protein in their organs.Science magazine reported on the new trial led by professor of medicine Julian Gillmore of University College London. Gillmore and her colleagues performed their trial at the Royal Free Hospital in England’s capital city, attempting to treat six patients for the rare genetic disease known as hereditary transthyretin amyloidosis (or TTR).

To treat the six patients, Gillmore’s team used a combination of CRISPR and mRNA technology. In essence, delivering the “clustered regularly interspaced short palindromic repeats”—a family of DNA sequences fund in the genomes of bacteria—via the same kind of lipid particles, or fat bubbles, that deliver the genetic codes for the spike proteins the COVID-19 vaccines use.

he doctors used the DNA sequences inside of fat bubbles to target mutated genes inside the patients’ liver cells. The malfunctioning genes churn out “misfolded” forms of the protein, transthyretin, which builds up on nerves and the heart; in turn leading to pain, numbness, and heart disease.

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