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A new paper, published today in Nature Medicine, lends weight to the idea that someday, we may be able to use a form of CRISPR-Cas9 gene editing, to treat genetic diseases in human children before they’re even born. The researchers who worked on the mouse study say they’re pleased with the results, but stress that the results are a far cry from being able to provide treatment to human fetuses.
Using a technique called “base editing,” researchers from the University of Pennsylvania School of Medicine and the Children’s Hospital of Philadelphia (CHOP) were able to treat mouse fetuses that had been engineered to have the gene mutation for a rare liver disease. This disease, hereditary tyrosinemia type 1 (HT1), begins to do damage before a fetus–whether mouse or human–is even born, says genetic engineering expert Kiran Musunuru, one of the study’s coauthors. Musunuru teaches at the University of Pennsylvania.
Decades - Disorder - Damage…in - Utero - Musunuru
“We’ve known for decades now that this is a liver disorder that already starts causing liver damage…in utero,” Musunuru says. Usually, this condition is treated with medication after birth, beginning as soon as it is detected. HT1 can cause liver cancer or even liver failure, along with a host of other symptoms. But in the mice, their new treatment impacts the disease earlier and only needs to be administered once–as opposed to the current (human) medication, which must be taken every day. Unlike the medication treatment, the gene editing treatment also means that a special low-protein diet isn’t needed.
Base editing is a technique that uses CRISPR Cas-9 technology, which turns a virus into a treatment carrier. Instead of sending a signal to the gene that cuts the DNA and enters new genetic code, like CRISPR Cas-9 does, with base editing the virus transports an enzyme to a specific point in the gene. That...
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