Skip to main content
Move the World.
crispr breakthroughs 2020

Lead Image © Andrew Brumagen

CRISPR, a tool for editing the genetic code in living organisms, was first discovered in 2007. Since then, the prospect of eliminating diseases by editing the human genome has been hovering on the edge of science fiction and reality.

Until 2020.

Here are four incredible CRISPR breakthroughs that happened this year.

Better Precision with a Tiny Protein

"When we think about how CRISPR will be applied in the future, that is really one of the most important bottlenecks to the field right now: delivery," Jennifer Doudna, who won the Nobel Prize in Chemistry along with Emmanuelle Charpentier in October for creating CRISPR, told Genetic Engineering News.

Accessing DNA in living cells has been the biggest challenge for CRISPR. The most widely used CRISPR gene editing system uses a protein called Cas9, which is naturally found in bacteria and archaea. Their biological role is to fight off viruses by destroying the viruses' DNA and cutting it out of their genomes.

However, scientists can use them as "DNA scissors," which can be used to cut disease-causing mutations out of any DNA.

The trouble is Cas9, as well as other proteins used in different CRISPR systems, are all really bulky, which makes it difficult to access the smallest nooks and crannies of the human genome.

However, in July Doudna and her University of California Berkeley team announced that they found a new Cas protein.

Drumroll, please. Enter the tiny CasΦ (pronounced "Cas-phi").

At practically half the size, CasΦ can reach areas of the human genome that most CRISPR gene editing proteins cannot. This new protein could be a game-changer for genetic engineering.

Gene Therapy Seen as a Cure for Sickle Cell Disease

This year researchers announced the results of an on-going clinical trial that tested CRISPR's ability to edit the DNA of living cells and possibly cure genetic diseases in humans. 

The study focused on two of the most common genetic disorders globally — sickle cell disease and beta-thalassemia. The only cure for these blood diseases is via a stem cell transplant from a viable donor. But often, the recipients have to take immunosuppressants for months or years, with difficult side effects. 

The trials involved collecting blood stem cells from the patients. Then, researchers used CRISPR to alter the cells' genes, and finally transplanted the stem cells back into the patients. Several months later, when patients reported that their symptoms were gone, the scientific community hailed the study as a gene therapy breakthrough — calling it a cure.

"I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease," researcher Haydar Frangoul said in a news release.

The next step is expanding the study by enrolling 45 patients and observing them long-term.

Gene-Edited Pigs Could Be Future Organ Donors

The U.S. doesn't have enough donor organs to fulfill every need. As a result, approximately 17 people die each day because they couldn't get the liver, heart, or lung they required. 

To solve this shortage, scientists have been looking into cross-species donation — or xenotransplantation — for a decade, with little progress. Our immune system is designed to attack intruders, which is excellent when you have the flu, but it makes xenotransplantation practically impossible. 

"The approach, if validated through further studies, could help solve global organ shortage (and) alleviate transplantation needs."

George Church

Now, thanks to CRISPR, xenotransplantation could be possible. Using CRISPR, researchers at Qihan Biotech modified the DNA of pigs to make them more compatible with humans. The researchers made 13 genetic modifications to the pigs, all in the hopes of making them more acceptable to the human body. In vitro lab tests showed promise — cells from the gene-edited pigs appeared less likely to be rejected by the human immune system than those of unmodified animals.

"The approach, if validated through further studies, could help solve global organ shortage (and) alleviate transplantation needs," Qihan Biotech co-founder George Church said in a press release.

Further studies are underway: the team is now testing an organ transplant from a gene-edited pig into a primate.

CRISPR Enters The Human Body for the First Time

Scientists at the Casey Eye Institute in Portland used CRISPR inside the human body for the first time — in a patient that had an inherited form of blindness.

Doctors dropped the gene-editing tool behind the retina via three drops of liquid that passed through a tube the size of a human hair. Once in the body, CRISPR went to work. It snipped the mutated gene on both sides of the problem area. They hope that once the mutation was removed, the snipped DNA will reconnect itself, allowing the gene to function as it should. They are now in clinical trials.

"Once the cell is edited, it's permanent and that cell will persist hopefully for the life of the patient," because these cells don't divide, said one study leader not involved in this first case, Dr. Eric Pierce at Massachusetts Eye and Ear.

If all goes according to plan, the patient should regain their vision within a few months. Next, the doctors plan to further test the procedure on 18 children and adults.

We'd love to hear from you! If you have a comment about this article or have a tip for a future Freethink story, please email us at [email protected]

Up Next

Clean Energy
Wind Turbine Blades Are a Growing Waste Problem
wind turbine blades
Clean Energy
Wind Turbine Blades Are a Growing Waste Problem
Using decommissioned wind turbine blades to build bridges, buildings, and more could eliminate one of the biggest disadvantages of wind energy.

Using decommissioned wind turbine blades to build bridges, buildings, and more could eliminate one of the biggest disadvantages of wind energy.

Hacking
Ethical Hacking Challenge: Can You Take Over a Military Satellite?
Ethical Hacking
Hacking
Ethical Hacking Challenge: Can You Take Over a Military Satellite?
The U.S. Air Force is hosting Hack-A-Sat, an ethical hacking competition challenging participants to find security vulnerabilities in satellite systems.

The U.S. Air Force is hosting Hack-A-Sat, an ethical hacking competition challenging participants to find security vulnerabilities in satellite systems.

Robotics
New Exosuit Helps Stroke Survivors Walk Farther and Faster
exosuit
Robotics
New Exosuit Helps Stroke Survivors Walk Farther and Faster
A new exosuit helps stroke survivors overcome hemiparesis by assisting them in making two key walking motions with their feet.

A new exosuit helps stroke survivors overcome hemiparesis by assisting them in making two key walking motions with their feet.

Dope Science
New Promise for Psychedelics and Depression
studying psychedelics and depression
Dope Science
New Promise for Psychedelics and Depression
New findings on psychedelics and depression show the benefits of microdosing, and could present more effective treatment options.
By Kurt Hackbarth

New findings on psychedelics and depression show the benefits of microdosing, and could present more effective treatment options.

Drones of the Future
This Nail Gun Wielding Drone Looks Terrifying, But Could Save Lives
This Nail Gun Wielding Drone Looks Terrifying, But Could Save Lives
Drones of the Future
This Nail Gun Wielding Drone Looks Terrifying, But Could Save Lives
According to OSHA, one in five workplace deaths in 2017 were in construction. Can nail gun drones help save lives?

According to OSHA, one in five workplace deaths in 2017 were in construction. Can nail gun drones help save lives?

Dispatches
A New Kind of Headset “Hears” Words You Don’t Say
A New Kind of Headset “Hears” Words You Don’t Say
Dispatches
A New Kind of Headset “Hears” Words You Don’t Say
The project, named AlterEgo, intentionally crosses the line between what's "out there" and what's in your head.

The project, named AlterEgo, intentionally crosses the line between what's "out there" and what's in your head.

Superhuman
These Gloves Can Teach You to Play the Piano. And Maybe Heal Your Brain.
These Gloves Can Teach You to Play the Piano. And Maybe Heal Your Brain.
Watch Now
Superhuman
These Gloves Can Teach You to Play the Piano. And Maybe Heal Your Brain.
Through "passive haptic learning", these gloves can teach you how to play the piano in an hour. Braille in four hours. Now researchers want to see if victims of traumatic brain injuries can use these gloves to re-learn critical skills.
Watch Now

Georgia Tech researchers Thad Starner and Caitlyn Seim have developed a pair of gloves for playing piano that can magically get you up to speed in just an hour. They've also taught blind people to read braille in four hours, a process that usually takes up to four months. The gloves work through a process called passive haptic learning, and is another great discovery from Georgia Tech researchers. Basically, they vibrate in...

Science
Bionic Prosthetic Grants Amputee Musician a Rocking Encore
Bionic Prosthetic Grants Amputee Musician a Rocking Encore
Science
Bionic Prosthetic Grants Amputee Musician a Rocking Encore
How might your life change if you lost an arm? After losing his right arm in an electrical accident, Jason wasn’t...
By Blake Snow

How might your life change if you lost an arm? After losing his right arm in an electrical accident, Jason wasn’t sure if he’d ever be able to drum again.

Could Growing Vaccines in Plants Save Lives?
Could Growing Vaccines in Plants Save Lives?
Watch Now
Could Growing Vaccines in Plants Save Lives?
Vaccines for influenza, polio, smallpox, even Ebola have all be grown … in plants.
Watch Now

This flu season has been nasty in large part because the vaccine didn’t work as well as past versions. So scientists like Professor George Lomonossoff of the John Innes Centre are on the hunt for new ways to make better vaccines and think they might have found one -- by growing them in plants.