New genome editing tools could fix 1000’s of diseases


Harvard and MIT scientists develop new tools to fix genetic mutations among the most likely to cause disorders from genetic deafness to cystic fibrosis, according to an article on

It says Harvard chemistry professor David Liu to journalists that ABE is almost like a pencil that can rewrite the letters that make up each strand of DNA so they’re arranged properly into the sentences that make up our genomes.

In the case of genetic mutations the “letters” are switched around, causing disorders like genetic blindness and cystic fibrosis. ABE makes it possible to reverse one specific mutation by putting those letters in the right order so they pair up correctly to form the right genetic “words” or “sentences.” This eliminates the genetic disorders that come from jumbled letters.

At the same time, a different lab at MIT and Harvard’s Broad Institute have developed a new Crispr-based system to edit the RNA in human cells instead of DNA, which holds promise for treating genetic disease.

According to the Britannica, the idea of using gene editing to treat disease or alter traits dates to at least the 1950’s and the discovery of the double-helix structure of DNA. In the mid-20th-century era of genetic discovery, researchers realized that the sequence of bases in DNA is passed (mostly) faithfully from parent to offspring and that small changes in the sequence can mean the difference between health and disease. Recognition of the latter led to the inescapable conjecture that with the identification of “molecular mistakes” that cause genetic diseases would come the means to fix those mistakes and thereby enable the prevention or reversal of disease. That notion was the fundamental idea behind gene therapy and from the 1980’s was seen as a holy grail in molecular genetics.

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