Hope for Progeria
Scientists have achieved a potential breakthrough in the fight against progeria, demonstrating that gene editing can fix a key mutation, effectively doubling the life span of diseased mice. The rare and incurable disease, which affects about one in 20 million people, causes children to rapidly age, with most dying from an associated condition in their teens.
The approach relies on a technique similar to CRISPR known as base editing (see the difference), which can modify pieces of genetic code without breaking the DNA backbone. In progeria patients, a single error in the genetic code limits production of a key protein that normally helps shape the cell nucleus. By fixing the error, normal production of the protein—known as lamin A—resumed and many progeria symptoms were alleviated. The achievement, which must be tested in humans, is a key advancement for similar genetic conditions where full CRISPR editing is not feasible.
Brush up on DNA and nucleotides here.
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