The GLP is committed to full transparency. Download and review our Annual Report.

Using CRISPR to build a ‘massive library’ of tools to cure genetic disease

| | June 21, 2018

In the past few years, [David] Liu’s become one of the most brightly-shining luminaries in the rapidly advancing field of gene editing.

Now he’s setting his custom-built evolution engines loose on the molecular machines that cut, paste, erase, and edit DNA. His goal is to create a massive library of disease-targeting tools—so that one day when scientists want to make a genetic fix, they can just pull whichever one they need off the shelf.

[A] “base editor” [was] a modified Cas9 enzyme that didn’t cut DNA. Instead, it worked more like a pencil, rewriting single nucleotides to convert C:G base pairs to T:A. That kind of a fix could cure about 15 percent of the 32,000 single base errors that cause genetic disease. [Researcher Nicole] Gaudelli wanted to go after a bigger piece of the pie. If she could make an editor that flipped A:T to G:C, that would address half those diseases.

Related article:  Genome surgeons target genetic disease at the source

Seven rounds and two grueling years later she had her new base editor. Liu submitted their paper describing a way to fix half the disease-causing single base snafus to Nature.

“Taxpayers don’t support our research so that we can just publish more papers,” he says. “We have an obligation to return these technologies to the public for the benefit of society.”

Read full, original post: Inside a chemist’s quest to hack evolution and cure genetic disease

The GLP aggregated and excerpted this article to reflect the diversity of news, opinion, and analysis. Click the link above to read the full, original article.
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.

Send this to a friend