ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects

We’ve been hearing A LOT about so-called ‘gene editing’ and ‘CRISPR’ lately — and now there’s new developments on that front.

Scientists have developed a new gene-editing technology called ‘prime editing’ that could potentially correct up to 89% of genetic defects, including those that cause rare diseases like sickle cell anemia and Tay Sachs disease.

This new and potentially game-changing technique was developed by researchers from the Broad Institute of MIT and Harvard, who published their findings in the journal Nature.
Let’s take a closer look!

What are ‘Gene Editing’ and ‘CRISPR’?

Genome editing is the alteration (aka editing) of a selected DNA sequence in a living cell.

Gene editing technologies enable scientists to make changes to DNA, leading to changes in physical traits, like eye color, and disease risk. Scientists use different technologies to do this. These technologies act like scissors, cutting the DNA at a specific spot. Then scientists can remove, add, or replace the DNA where it was cut.

CRISPR is one of those technologies.

CRISPR (clustered regularly interspaced palindromic repeats) allows scientists to make changes to the DNA of living organisms more precisely and inexpensively than before.

Think of CRISPR as the “find and replace” function; however, CRISPR sometimes mis-recognizes a DNA sequence that is similar to the one it’s looking for and cuts in the wrong place, causing “off-target mutations.” Other times it might cut in the right place, but cause mistakes, or “indels,” where DNA is incorrectly inserted or deleted.

So in other words, it’s a work in progress — but a GREAT progress nonetheless. 

‘Prime Editing’

Prime editing builds on CRISPR, but is more precise and versatile — directly writing new genetic information into a specified DNA site, according to researchers.
Prime editing combines the CRISPR technique with a different protein that can generate new DNA. The tool nicks the DNA strand, then transfers an edited sequence to the target DNA — allowing researchers to more easily insert and delete parts of human cells.
“With prime editing, we can now directly correct the sickle-cell anemia mutation back to the normal sequence and remove the four extra DNA bases that cause Tay Sachs disease, without cutting DNA entirely or needing DNA templates,” said David Liu, one of the authors of the study, in a press release.
The team of researchers will now continue working to hone the technique, trying to maximize its efficiency.
They will also continue testing on different models of diseases to ultimately “provide a potential path for human therapeutic applications,” according to the press release.
Seems like a watershed moment is on the horizon!
We’ll keep a close eye on any developments. 

 


What are your opinions on or experiences with gene editing? Share your stories, thoughts, and hopes with the Patient Worthy community!

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