Mass Spectrometry & Spectroscopy
What Is Chemical Surgery?
Oct 17 2017
Currently, around two-thirds of known human diseases are point mutations caused by genetic variants. So what if medical scientists could reprogram genetic codes to eliminate imperfections and remove disease?
Introduced to the world as "chemical surgery," a team of Chinese researchers have mastered the concept and successfully performed the procedure on human embryos. The feat is a world first and could spell exciting new advancements for the medical sphere.
Base editing used to cancel out beta-thalassemia
Working out of Sun Yat-sen University, the team used a technique called base editing to correct a single error pinpointed in a genetic code sequence made up of three billion "letters." They worked with lab-made embryos and used the technique to effectively remove a potentially life-threatening blood disorder called beta-thalassemia. According to the team, the approach could one day be used treat a host of inherited diseases.
So how does the complex process work?
Cut, copy and paste
Basically, genetic editing alters the fundamental building blocks of DNA. They're called adenine, cytosine, guanine and thymine, though are commonly known by their respective letters, A, C, G and T. Together the quartet contains all the instructions needed to build and run the human body.
Beta-thalassemia was the perfect disease to work with as it's triggered by a single change to a base in the genetic code. This is known as point mutation, which the Chinese team were miraculously able to correct by scanning DNA for the error, then converting the erroneous G to an A.
"We are the first to demonstrate the feasibility of curing genetic disease in human embryos by base editor system," explains Junjiu Huang, one of the researchers.
According to Huang and his colleagues, the study opens new doors for treating patients and preventing babies from being born with beta-thalassemia. It could also revolutionise treatment for other inherited diseases.
The genetics revolution
While it's an exciting step forward, the concept isn't entirely new. Base editing is an advance on an existing form of gene-editing called Crispr. The concept is revolutionising science and empowering researchers with the ability to break DNA and insert new genetic information.
Though unlike Crispr, "chemical surgery" works directly on the DNA bases themselves. As a result, the technique is more efficient and has less unwanted side-effects.
For scientists like Huang, pushing the boundaries of medical science is just another day at the office. For a closer look at the latest developments, 'Pushing the Limits of Speed and Sensitivity in Drug Screening – an LC-MS solution' spotlights Toxtyper™, a revolutionary automated drug testing solution from Bruker.
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