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 9:40 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.

Mississippi Coronavirus Cases

Data is updated nightly.

Cases: 314710

Reported Deaths: 7254
CountyCasesDeaths
DeSoto21646260
Hinds20369416
Harrison17949309
Rankin13643278
Jackson13450246
Madison10113217
Lee9986174
Jones8384163
Forrest7689152
Lauderdale7198240
Lowndes6403148
Lamar623686
Lafayette6203119
Washington5341134
Bolivar4802132
Oktibbeha462998
Panola4596107
Pearl River4519146
Marshall4450103
Warren4393121
Pontotoc420872
Monroe4115133
Union411176
Neshoba4031176
Lincoln3969110
Hancock379586
Leflore3498125
Sunflower336290
Tate334784
Pike3327105
Scott316274
Alcorn313368
Yazoo311770
Itawamba300577
Copiah297465
Coahoma295579
Simpson295388
Tippah288768
Adams286982
Prentiss280060
Marion269380
Leake268473
Wayne262841
Grenada261587
Covington259881
George248148
Newton246862
Winston227581
Tishomingo227067
Jasper221148
Attala214473
Chickasaw208057
Holmes189174
Clay185554
Stone182833
Tallahatchie178941
Clarke178080
Calhoun170932
Yalobusha164638
Smith162534
Walthall134245
Greene130633
Lawrence128724
Montgomery127142
Noxubee126734
Perry126338
Amite123142
Carroll121829
Webster114532
Jefferson Davis107133
Tunica105726
Claiborne102430
Benton100025
Humphreys96733
Kemper95828
Franklin83923
Quitman81116
Choctaw76418
Wilkinson67531
Jefferson65728
Sharkey50217
Issaquena1686
Unassigned00

Alabama Coronavirus Cases

Cases: 537813

Reported Deaths: 11024
CountyCasesDeaths
Jefferson791691529
Mobile41177808
Madison35002507
Tuscaloosa25871454
Shelby25076249
Montgomery24549591
Baldwin21290309
Lee15946171
Calhoun14556319
Morgan14364280
Etowah13890353
Marshall12262223
Houston10602282
Elmore10115206
Limestone10031151
St. Clair9890245
Cullman9730194
Lauderdale9449243
DeKalb8853188
Talladega8325176
Walker7259277
Autauga6971108
Jackson6830112
Blount6750139
Colbert6317134
Coffee5546119
Dale4869113
Russell444338
Chilton4343113
Franklin426282
Covington4138118
Tallapoosa4040152
Escambia394577
Chambers3581123
Dallas3564153
Clarke351361
Marion3137101
Pike311977
Lawrence302298
Winston275673
Bibb263064
Geneva252577
Marengo249664
Pickens234862
Barbour231956
Hale223677
Butler217869
Fayette212462
Henry189644
Cherokee184345
Randolph182042
Monroe178140
Washington167639
Macon160750
Clay156957
Crenshaw153357
Cleburne149241
Lamar143035
Lowndes139653
Wilcox127430
Bullock123041
Conecuh110629
Coosa108928
Perry107826
Sumter104932
Greene92634
Choctaw61024
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