The most effective gene-editing tool available today is CRISPR-Cas9. Just this year, scientists have successfully used it for a wide variety of experiments, from modifying vegetables to encoding a Gif in bacterial DNA. However, most recently, it was used to remove a genetic disease from a human embryo.
Although extremely powerful, CRISPR-Cas9 does have its limitations; it’d only able to target DNA. Therefore, to extend its capabilities to RNA editing, scientists from the University of California San Diego School of Medicine developed a modification of CRISPR, and they’ve called it RNA-targeting Cas9 (RCas9).
In a study published in Cell, the UCDS team tested their new tool by correcting the kinds of molecular errors that cause people to develop microsatellite repeat expansion diseases, such as hereditary amyotrophic lateral sclerosis (ALS) and Huntington’s disease.
During standard CRISPR-Cas9 gene editing, a “guide” RMA is instructed to deliver a Cas9 enzyme to a specific DNA molecule. However, the scientists from UCSD instructed it to target an RNA molecule instead.
Tests that were conducted in the laboratory showed that RCas9 removed 95% of problem-causing RNA for myotonic dystrophy types 1 and 2, Huntington’s disease, and one type of ALS. This technique also reversed 93% of the dysfunctional RNA targets in the muscle cells of patient with myotonic dystrophy type 1, resulting in healthier cells.
Senior author Gene Yeo, a cellular and molecular medicine professor at UCSD School of Medicine, explained that “this is exciting because we’re not only targeting the root cause of diseases for which there are no current therapies to delay progression, but we’ve re-engineered the CRISPR-Cas9 system in a way that’s feasible to deliver it to specific tissues via a viral vector.
Across the world, an estimated 450,000 people are living with ALS. Roughly 30,000 of these are American, where 5,600 people are diagnosed with the disease each year. However, the exact number of Huntington’s disease cases is as easy to pin down. One estimate suggests that around 30,000 Americans display symptoms of it, while more than 200,000 are at risk.
Regardless of the exact figures, these two neurological diseases clearly affect a large number of people. This prevalence and the absence of a known cure makes the UCSD team’s research all the more relevant. Even more exciting is the fact that some kinds of RNA mutations that this study is targeting are known to cause over 20 other genetic diseases.
However, the researchers are well aware that what they have accomplished so far is just the first step. While RCas9 works in labs, they still have to figure out how it will fare when tested on humans.
Yeo explains that “the main thing we don’t know yet is whether or not the viral vectors that deliver
RCas9 to cells would elicit an immune response. Before this could be tested in humans, we would need to test it in animal models, determine potential toxicities, and evaluate long-term exposure.”
Ultimately, while RCas9 couldn’t deliver a cure exactly, it could potentially extend a patient’s life. For diseases such as ALS and Huntington’s, that’s a very good place to start.
Source: futurism
Images: depositphotos
Why Do Cats Like Catnip?
Are they really getting "high," or is there scientific logic behind cats' love of catnip?
Winter Hikers, Gather Round for the Best Tip of the Season
Here's how advanced snow hikers layer clothing in the winter.
Was There a Time a Day Lasted 10 Hours?
Eons ago, before the dinosaurs ever evolved, there was primordial life on Earth. What did it look like?
You Won't Believe What the Human Body Does in One Minute
How much do you think it is capable of doing in a single minute? Watch this video and you'll have your answer!
These Insane Diseases Have Baffled Scientists for Years
Medical science has come a long way, but there are still a number of diseases that science cannot explain. Here are some of them:
9 Inspirational Female Scientists You Should Know About
History is full of women who made significant contributions to science. Let’s meet the most influential ones.
I Never Knew These Stunning Facts About Our Universe
If you thought you understood the universe, you'll be completely floored by this insane facts.
These 20 Tech Inventions Will Define the Next Decade
The future is here. Check out some emerging technologies that are all set to chane our world forever.
Incredible Science Breakthroughs That Made News in 2022
Let’s check out some of the most interesting scientific discoveries of 2022.
Are Smartphones Making Us Stupid? This Will Surprise You!
If you use a smartphone you need to see this!
These Will Give You True Perspective About Our World
Just to give you an idea of how big the Earth really is in true perspective to all the other heavenly bodies up there in the cosmos.
Is There Any Way To Destroy or Deflect Asteroids?
Watch an interview with Cathy Plesko, a leading scientist working on preparing for the dangers of an asteroid attack
The Story of the Internet: Who REALLY Invented It?
Who really invented the internet, and why did they REALLY do it?
17 Incredible Eye Facts You Have To See To Believe
They say the eyes are the window to the soul, and they also happen to be our windows to the world. Here are 17 insanely fascinating facts about eyes.
15 Frightening Animals Early Humans Had to Live With
10 scary animals early cavemen in Africa had to deal with 200,000 years ago.
Six Inventions Da-Vinci Made Ahead of Their Time
He created and envisioned many inventions that were sometimes even centuries ahead of their time. Was there ever a genius as great as Da-Vinci?
Is It 3024 Already?! – 14 Wildly Futuristic Things
These revolutionary ideas are about to change our world.
The First Robot Ever Made Was Created 250 Years Ago...
This automaton is pure genius, especially when you consider it was built 250 years ago...
TRIVIA: 25 Shocking Differences Between the Sexes
Did you know that there are some vast differences between the sexes? These 25 scientific facts will probably come as a big surprise to you, they did to me.
These Expensive Megaprojects Proved to be a Waste of Time
Not all megaprojects are useful. Here are some worthless ones.
Fascinating: What is the Deepest Hole Humanity Has Dug?
This video explains how low humanity has dug down so far.
First US Embryo to be 'Edited'; Rise of Genetic Changes!
Could the first human embryos be genetically modified?
7 Exciting Physics Tricks You Have to See!
Watch these seven remarkable physics tricks in this exciting and fun video.
8 Inventions of 2023 That Will Change The Way We Live
This year was a prominent one for ground-breaking innovations that will change the way we live.
What is Your Consciousness? A Remarkable Video...
This video by Ted-ed searches to explain the origin of consciousness as well as what is consciousness.
Are You a Late Sleeper? This Super Video Explains Why
Why is it that some people are late risers, and some are morning people? It's all down to our genetics. Have a look here to find out more.
Wow! Who Knew That Jupiter Was So Mesmerizing?
Take a look at Jupiter like you've never seen it before!
These 11 Brain Facts Are Enough to Boggle Your Mind
When you consider the sheer complexity of the human brain, it's difficult to wrap your head around it. Learn these 11 fascinating facts about the human brain.
The History of Our Planet in One Beautiful Spiral...
This beautiful infographic offers us a remarkable story – the history of Planet Earth.
13 Incredible ROOF INNOVATIONS For Your House
Keep the roof of your house safe with these incredibly useful inventions.
Remembering the Life and Wisdom of Stephen Hawking
Stephen Hawking possessed one of the greatest minds the world has ever seen, but was also impaired by a debilitating disease. Here are his life ant times.
10 New Things We've Learned About The Brain
We have learned some astonishing new things about the human brain in recent times.
Do You Know What Alzheimer's Really Is?
This video will shed light on the different stages of this complex and incredibly destructive disease.
This Man Built an Automatic Train System for Trash Cans!
Wouldn’t it be cool if a robot picked up and moved our garbage out for pickup with the push of a button?
8 Biggest Mysteries Still Surrounding the Human Body
Although modern science has revealed much about the human body, there are some things that still remain unknown. Here are the 8 biggest mysteries about it.
What’s it Like Using a 115-Inch TV?!
This colossal 115-Inch TV will transform your viewing experience forever.
How Can You Recognize a Fake Gadget Before Buying It?
More often than not, you're almost unable to recognize them. So, before purchasing the product you want to have, bear in mind the following tips:
Discover How Anesthesia Affects Your Body and Mind
If you were ever curious about how exactly anesthesia works, then this video has all the answers!
Breathalyzers May Be More Important Than You Thought
This video does a great job in breaking down how in the future, breath analysis can be used to detect disease and prevent more invasive procedures.
When Under a Microscope, Even Ordinary Things Seem Alien
Have you ever given any thought to what every day things look like under a microscope? Some of these images are truly beautiful - see for yourself in the images below.
This Giant Tank Will Unlock the Universe’s Big Mysteries
Japan is building a huge underground tank inside a mountain to help unlock the many mysteries of the universe.
19 Everyday Items That Started as Military Inventions
Look around your home, and you’ll surely find a handful of these items that were originally developed for the armed forces.
Travel From the Sun to Pluto With Our Interactive Guide!
The solar system is a fascinating place, and we wanted to bring you an interactive journey that you can use to travel through it. Enjoy the journey!
The Curious Science of Tricking Your Taste Buds
Did you know you could confuse your sense of taste? Find out how!
GUIDE: How Alcohol Affects Us, Drink by Drink...
In this article, we're going to discuss the exact amounts of alcohol in our blood and what they look like, including how each level affects us and our ability to function.
25 Feats of Engineering that Leave Everyone Amazed
The amount of planning and sheer brain power it took to design and execute these structures, proving once again that there are true geniuses out there.
10 Incredible Animal-Inspired Robots
Let's meet some incredibly realistic robotic animals.