New gene editing technology could correct 89% of genetic defects

Scientists have developed a new gene-editing technology that could potentially correct up to 89% of genetic defects, including those that cause diseases like...

Posted: Oct 22, 2019 7:40 AM

Scientists have developed a new gene-editing technology that could potentially correct up to 89% of genetic defects, including those that cause diseases like sickle cell anemia.

The new technique is called "prime editing," and was developed by researchers from the Broad Institute of MIT and Harvard, who published their findings Monday in the journal Nature.

Prime editing builds on powerful CRISPR gene editing, but is more precise and versatile -- it "directly writes new genetic information into a specified DNA site," according to the paper.

In the traditional CRISPR-Cas9 approach, Cas9, a type of modified protein, acts like a pair of scissors that can snip parts of DNA strands. It can target genes in a specific location -- for instance, to disrupt a mutation.

About two-thirds of known human genetic variants associated with diseases are single point gene mutations, so gene editing has the potential to correct or reproduce such mutations.

Prime editing combines the CRISPR-Cas9 method 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 smoothly insert and delete parts of human cells.

The technique allows researchers to search and replace entire sections of DNA strands, all without disruptive breaks or donor DNA. With this method, researchers say they hope to accurately and efficiently correct up to 89% of known disease-causing genetic variations.

"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 Broad Institute press release.

"The versatility of prime editing quickly became apparent as we developed this technology," said Andrew Anzalone, another author in the study, in the press release. "The fact that we could directly copy new genetic information into a target site was a revelation. We were really excited."

The team of researchers will now continue working to hone the technique, trying to maximize its efficiency in various cell types and exploring any potential effects on the cells. 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.

Gene editing is still a relatively young and rapidly expanding field of study -- CRISPR-Cas9 is based on a decade-old discovery, but was only used on humans for the first time in 2016. Then in 2017, the Broad Institute developed a new technique called base editing, which can make changes to a targeted DNA site without cutting the DNA.

Researchers at the Broad Institute and elsewhere hope CRISPR could one day target a wide range of "bad" genes -- potentially helping humans avoid obesity, Alzheimer's disease, genetic forms of deafness, and more.

However, as the technology has advanced, doctors, scientists, and bioethicists have also raised ethical questions. Some fear it could open the door to human embryos being manipulated for nontherapeutic reasons, or that it could create unintended mutations and new diseases.

Just earlier this year in March, a group of researchers, including the scientist who pioneered and patented CRISPR technology, called for a global moratorium on human germline editing -- changes made to inherited DNA that can be passed on to the next generation.

They listed ethical concerns, and pointed to Chinese scientist He Jiankui, who claimed to have made gene edits when creating two AIDS-resistant babies last year. He's work, which could have unforeseen consequences, has been internationally condemned and called "abominable in nature" by Chinese authorities.

California Coronavirus Cases

Data is updated nightly.

Cases: 3573549

Reported Deaths: 52491
CountyCasesDeaths
Los Angeles119295421467
Riverside2897733792
San Bernardino2867552940
Orange2614083921
San Diego2606253303
Santa Clara1107551781
Kern103422877
Fresno955481443
Sacramento935281484
Alameda807771242
Ventura77749852
San Joaquin668291126
Contra Costa62720681
Stanislaus56235949
Tulare48013761
Monterey42261328
San Mateo39059515
San Francisco34291422
Santa Barbara32050413
Solano30115164
Merced29147401
Sonoma28193298
Imperial26909635
Kings22082220
Placer19861232
San Luis Obispo19696235
Madera15496214
Santa Cruz14671183
Marin13231197
Yolo12851185
Shasta11017174
Butte10970163
El Dorado9160100
Napa905369
Sutter888097
Yuba577337
San Benito577261
Lassen562419
Tehama508652
Nevada397274
Tuolumne396059
Mendocino381643
Amador346641
Humboldt321033
Lake316441
Glenn223023
Colusa213613
Calaveras191049
Siskiyou176014
Inyo131337
Mono12144
Del Norte10035
Plumas6536
Modoc4564
Mariposa3957
Trinity3705
Sierra1000
Alpine820
Unassigned00
Chico
Clear
43° wxIcon
Hi: 74° Lo: 37°
Feels Like: 43°
Oroville
Clear
49° wxIcon
Hi: 71° Lo: 49°
Feels Like: 49°
Chico
Clear
43° wxIcon
Hi: 66° Lo: 46°
Feels Like: 43°
Red Bluff
Clear
42° wxIcon
Hi: 53° Lo: 16°
Feels Like: 38°
Red Bluff
Clear
42° wxIcon
Hi: 70° Lo: 41°
Feels Like: 38°
Chico
Clear
43° wxIcon
Hi: 76° Lo: 40°
Feels Like: 43°
We've had beautiful weather over northern California today and it will continue through Wednesday before changes begin. Those changes will first involve cooler air and stronger wind, with rain and snow chances following.
KHSL Severe
KHSL Radar
KHSL Temperatures

Community Events