What is CRISPR? The Life-Changing Science Behind CRISPR

What the heck is CRISPR and why did two women win a 2020 Nobel prize in chemistry for an acronym? CRISPR is a game changer. CRISPR is science that will change lives.

WHAT IS CRISPR? A GENETIC SCISSORS

CRISPR is a genetic scissors.  No not Louie’s scissors! (which I had to take away, he shouldn’t be playing with scissors)

The term CRISPR comes from ‘clustered regularly interspaced short palindromic repeats

That’s a mouthful. No wonder they came up with an acronym.

 

CRISPR genetic scissors and Louie
CRISPR is a genetic scissors...okay not this scissors

PALINDROMES

Palindromes are words, phrases, or sentences that repeat backwards or forwards, such as ‘Madam, I’m Adam.” Or the date 02/02/2020.

So the term ‘…palindromic repeat’ is –technically–redundant.

There will be two palindrome weeks in 2021.

The first begins on 1/20/21 and will continue until 1/29/21 1/21/21, 1/22/21,1/23/21,1/24/21 ... 1/29/21!

This hysteria begins again on 12/1/21!

Louie can’t wait!

Okay, so CRISPR is a genetic scissors. 

What are we cutting with the genetic scissors?

WHAT ARE GENES? WHY DO WE WANT TO CUT GENES?

Genes are a section of DNA. Long strands of DNA with lots of genes make up chromosomes.

Any given gene section along the DNA can be thought of as an instruction manual.

The gene sections have instructions encoded. The instructions allow any given cell to produce a specific product – usually a protein, such as an enzyme – that triggers one precise action.

THE SCIENCE BEHOND CRISPR
DNA molecule

Sometimes, these gene sections go haywire, and instruct the cells to make a product that does not help the body.  Enter genetic diseases.

We need genetic disease like we need extra toes. We can cut off an extra toe. So why not cut –or silence– the gene section that isn’t doing us any good? Then our cells will stop recreating the errors.

sixth toe radiograph
Count the toes...

WHAT IS CRISPR AND DNA

Lucky for us, the DNA molecule has been sequenced! We can read the cell instruction manual.

In DNA sequencing, we are determining the order of nucleotides in a strand of DNA.  Nucleotides are the molecules or building blocks of DNA. (pic on right)

Every organism’s DNA consists of a unique sequence of nucleotides.

Many amazing scientists have contributed to biochemistry and genetics. There will be many more! 

base pairs hold DNA strand together

To find ways to treat diseases at the genetic level, is to edit them. Sorry, I sound like Yoda now.

yoda
Yoda
DNA building blocks ATGC
DNA code

WHAT IS GENE EDITING?

First, researchers need to modify genes in cells, so they can ascertain which segment on the DNA causes a specific disease, or which segment on a plant makes it so tasty to insects. They can’t change the gene code until they know what the code is.

This used to be time-consuming, difficult and sometimes impossible work.

THE GOAL OF GENE EDITING
TO DISRUPT, SILENCE or INACTIVATE GENETIC MATERIAL
OR TO CORRECT, INSERT or REPLACE GENETIC MATERIAL
Our genome contains 25,000 protein-encoding genes
Some diseases, such as Phenylketonuria, Huntington disease, and Sickle Cell anemia are caused by a SINGLE gene.
More info here
Previous
Next

2020 NOBEL PRIZE IN CHEMISTRY : Cas9 Genetic Scissors

Enter…CRISPR!

In 2012, Emmanuelle Charpentier and Jennifer A. Doudna collaborated. They discovered one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants, and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies, and may make the dream of curing inherited diseases come true.

nobel prize winners chemistry 2020
© Johan Jarnestad/The Royal Swedish Academy of Sciences

CRISPR IS LIFE-CHANGING!

No wonder they were awarded a Nobel Prize.

Using the CRISPR/Cas9 genetic scissors, it is now possible to change the code of life (genes, made up of those nucleotides above) over the course of a few weeks.

CRISPR: HOW DOES IT WORK?

The genetic scissors can be used to edit a cell’s genome with incredible precision – for example, it can cut out a dysfunctional gene associated with a hereditary illness. 

And if the healthy version of the gene is also delivered to the cell, the cell’s own repair system will then incorporate the healthy strands right where it was cut. Forevermore the cell with replicate the healthy code. It has a new instruction manual!

CRISPR genetic scissors © Johan Jarnestad/The Royal Swedish Academy of Sciences[/caption]

how crispr works courtesy UC Berkley[/caption]

HOW DOES CRISPR GET INTO THE CELL TO CHANGE IT?

With something called a vector.

A common delivery system is…a virus! I know, seems so strange, but as we all know, viruses are excellent at getting into our cells and wreaking havoc. Scientists use only a portion of the virus –the same way they do for many vaccines <for more on the SCIENCE OF VACCINES, I have a great blog post here!> so it’s perfectly safe and the virus doesn’t cause us to become infected.

AAV is commonly used as a delivery system. It was first discovered in 1965! Big milestone! 

AAV is now modified so it can get inside many different types of cells: liver, kidney, eyes, and the central nervous system.

Crossing the blood brain barrier (getting the vector into our central nervous system) is a major advancement toward the treatment of genetic diseases with gene replacement therapy.

WHAT IS CRISPR? SCIENCE THAT WILL CHANGE LIVES

I’m sure you realize now just exactly how fantastic it is to precisely speed up gene editing.

 In theory, CRISPR would be able to correct diseases such as sickle cell anemia. (remember SSA is caused by a single base change /one gene gone wrong.)

Think of it like this: A technician would take (bone marrow) cells out of your body. (Bone marrow is where red blood cells are made.)

The cells would be be modified in the lab, and then put back into you. Your DNA would no longer make the sickle cells but produce normal red blood cells.

DNA molecule genetic modification
CRISPR science that will change lives

You’d be cured of that disease forever and ever—all because of a procedure you went through when you were five years old.  You’re done, you’re fixed. 

CRISPR & WORLD HUNGER

The ability to cut any DNA molecule at a chosen site has huge potential – from treating genetic illnesses

to creating disease-resistant crops and increase crop yields

to making beer more cheaply or alter dairy products so they won’t spoil as quickly. 

rice plant
Disease-resistant rice
genetically altered hops for beer
Economical hops
Cheese
Cheese with a longer shelf life

So CRISPR is not just about curing human diseases! It’s going to help combat world hunger. (maybe not the beer part…)

Man is an enemy of monarchs
Feeding the world

CRISPER FEARS

Of course there has to be a negative…

In 2018, Chinese scientist He Jiankui revealed he had helped make the world’s first gene-edited babies. His goal was to engineer resistance to infection with the AIDS virus. His work was denounced as unsafe human experimentation, and he has been sentenced to prison in China.

That can’t be fun.

The CRISPR editing I’ve discussed is done after birth, to an individual, or to crops for a higher yield, or to make food less susceptible to bacteria.

Scientists fear CRISPR will be misused to make “designer babies” via altering eggs, embryos, or sperm. These are changes that can be passed on to future generations.

mighty mouse
Supermice will rebel and never be test subjects again

Louie needs no gene therapy. He’s perfect! But if he manifested a genetic disease, he’d have CRISPR.

CRISPR dog
All dogs go to heaven

GENE THERAPY : A GAME CHANGER

Sure, we could write another dystopian novel about all this. In fact, I might! This science is cool and amazing. Magical!

The CRISPR/Cas9 genetic scissors has taken botany, biology, biochemistry, and genetics into a new epoch and can bring great benefits to humankind.

Trials have even shown how CRISPR delivered genetic modifications can spread through populations of mosquitoes and stop malaria infections! Imagine eliminating malaria, the way we’ve done with smallpox.  

(For more on the science of vaccines, I’ve got a great post here!)

mosquito
Yuck

CRISPR : THE SCIENCE IS IN ACTION

And in the face of the Covid-19 pandemic, researchers have found ways to use CRISPR in rapid coronavirus diagnostic tests. Proposals have been made to use it to attack the virus’s genome.

CRISPR is used to treat acute lymphoblastic leukemia. 

In 2017, the FDA approved another gene therapy for an inherited form of vision loss!

More about the science of vision here...
Click Here

More than 2,600 gene therapy clinical trials are ongoing, have been completed, or approved worldwide.

Now that you may know more about the science behind CRISPR and the Cas9 genetic scissors, what do you think about it?

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58 thoughts on “What is CRISPR? The Life-Changing Science Behind CRISPR”

  1. This is a seriously important medical advance. Have seen the pain and suffering in people suffering with sickle cell disease. Congratulations to the two women scientists. I think it’s probably too late but I wish I could’ve been used to make me taller. Maybe in another lifetime. Mike

    Reply
  2. This is seriously good writing about a topic that I have already read quite a bit about lately, but nothing this concise and thorough. Good job.

    Reply
  3. I’ve read about people who have tried to gene hack themselves using this method and it didn’t seem to work, so I guess there’s still a lot of work to be done. To get around that issues, didn’t a Chinese scientist gene hack two embryos, causing a huge outrage?

    Reply
    • Yes, the scientist got into a lot of trouble and is serving a prison term. The science is amazing and far more accessible so there will be issues. I even found an online website selling CRISPR and vectors to anyone! I was tempted to proceed, just to see what would come in the mail. HA Thanks.

      Reply
  4. Hi Sue,

    This was such an amazing read! It’s mind-boggling to think that we have the technology to eliminate inherited disease, produce disease-resistant crops and so much more!

    I just hope no one else decides to use it for unsafe human experimentation. That is a worrying thought!

    I had to laugh at the photo of Louie with his “genetic scissors”. 😂

    Thanks for the informative post!

    Bianca xx | missmonro.com

    Reply
    • I’m also amazed with this important science! Imagine disease or drought resistant crops!? And eliminating inherited disease! Yes, Louie isn’t quite ready to participate just yet! Ha Thanks.

      Reply
  5. Thanks so much for writing about this Sue! I hadn’t had the chance to read up on it yet. Very ground breaking stuff although I will admit that when I heard the nobel prize was for gene editing I totally thought of all those sci-fi movies. 🤦🏻‍♀️🤷‍♀️

    Reply
  6. Sometimes you read something and think no way, that’s just science fiction. So to find out that this is real, going on today, and all the massive potential benefits is just mind blowing. I’ve never heard of CRISPR but no wonder they won the Nobel Prize! Fascinating read.

    Reply
  7. Hey Sue, you raise many positives regarding crispr in this blog post, but I think there are many ethical concerns behind the technology as well.

    There are many hypothetical scenarios we have yet to untangle and create institutional safeguards for. For instance, if a baby is born mutated because of crispr, how do we move forward? Who is responsible for the baby’s upbringing? If we detect anomalies in a gene-edited fetus, do we allow the baby to come to term, or do we ‘discard’ the baby as a failed experiment?

    It would be great if you can write a post that delves more into the ethical concerns of crispr. THAT would make for an excellent and thought-provoking read. (drop me a DM or an email if you do, I would love to read it!)

    Nevertheless, this blog post is excellent for anyone who has no clue what crispr is. Thanks for sharing!

    Reply
    • Great idea about the ethical concerns! Right now, no one would approve genetically altering fetuses –look at that scientist from China–but what if they did? We’d have super heroes or maybe super villains. It would make for a fun post, thanks for the idea!

      Reply
  8. This sound amazing. I hope this will be used to cure diseases and also Corona virus. Congratulations to the two women who worked so hard and Thanks to you, for sharing this amazing news… In the midst of chaos this is some hope to look forward to. ❤️

    Reply
  9. Fascinating, I knew about this but not the full details about how it works or the development. It is worrying if you think about the potential damage it could cause too though x

    Sophie

    Reply
  10. So informative! I learned so much from this post, it’s filled with lots of great information. I don’t think I’ve heard of CRISPR before but wow what a creation

    Reply
  11. I never would have searched for information on this on my own, but I am so glad I came across your post. This is such an interesting topic. My brain is swimming with all of the possibilities this new technology might bring in the future.

    Reply
  12. I’ve never even heard of CRISPR, a mouthful is an understatement haha! I absolutely love science that so complex like this, it really is just mind-blowing to read about, especially when I don’t know much about the field. Great post and thanks for sharing!

    Anika | chaptersofmay.com

    Reply

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Susan Berk Koch
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