New method of locating and targeting mutations promises “letter by letter” genome editing

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Beating-heart cells derived in which a single DNA base-pair of the PRKAG2 gene was edited using the method developed the Gladstone team. (CREDIT: Luke Judge/Gladstone Institutes, via GEN)

No matter how precise and powerful modern genetic modification techniques become, they’re useless in the battle against genetic disease if we don’t have a similarly precise and powerful way to identify the mutations that cause these diseases. This is the problem researchers at the Gladstone Institutes claim to have solved, according to Genetic Engineering and Biotechnology News:

“For our method to work, we needed to find a way to identify a single mutation among hundreds of normal, healthy cells,” said Gladstone research scientist Yuichiro Miyaoka, Ph.D., the paper’s lead author. “So we designed a special fluorescent probe that would distinguish the mutated sequence from the original sequences. We were then able to sort through both sets of sequences and detect mutant cells—even when they made up as little one in every thousand cells. This is a level of sensitivity more than one hundred times greater than traditional methods.”

With this new technique and its increased sensitivity, GEN writes that the Gladstone team has “found a way to edit the human genome one letter at a time. They add that this finding will not only boost the ability to model human disease, but will also pave the way for improved gene therapy approaches.”

Read the full, original article here: Editing the Human Genome One Letter at a Time

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