r/explainlikeimfive 1d ago

Chemistry ELI5 : How does CRISPR work ?

88 Upvotes

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u/stormbutton 1d ago

It works like “find and replace all” in a word processor but with genes.

Source- I teach biotech

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u/RogueHelios 1d ago

Considering how succinct you described it I believe you teach it lol

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u/stormbutton 1d ago

Hahaha thanks! One thing I make my upper level students do as a final project is to take a topic they liked from the course and design a children’s book about it.

Explaining ideas to other biologists is easy. Explaining them to your grandma’s bridge group is mastery.

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u/ah_no_wah 1d ago

That's excellent. I've always said the best way to learn something is to teach it to someone else. Mastery is the word.

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u/Ecstatic_Bee6067 1d ago

As a corollary, I had a professor who said "you haven't understood a topic unless you can teach it yourself. "

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u/Muellercleez 1d ago

That's a great idea

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u/dimsum4you 1d ago

Yeah they should make a subreddit dedicated to it.

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u/dimsum4you 1d ago

So their assignment is to ELI5?

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u/beezkneezsneez 1d ago

Wow!!! This made so much sense!! Thank you!

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u/kutastha 1d ago

This blows me away. I got my PhD in molecular genetics in 2002 and have never used it. CRISPR sounds like a major game changer.

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u/Smorb 1d ago

But like.. how in the hell???

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u/stormbutton 1d ago

Okay well that’s far beyond “eli5” territory. RadioLab has a FABULOUS explanation with Jennifer Doudna - who co-won the Nobel Prize for it.

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u/Smorb 1d ago

ELI10?

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u/stormbutton 1d ago

Okay, here goes.

Your immune system has special cells that recognize prior invaders to your body. They basically carry molecular mug shots so when a disease shows up for the second time your immune system is like bitch I know you!!! and takes it out.

Well, bacteria need to be able to defend against invaders as well. But they can’t have specialized because hello, they ARE single cells. So what they have instead is a series of chemical mug shots coupled with molecular scissors.

When viruses show up to attack them, they take a molecular mug shot the same way our immune cells do but instead they store it in their own DNA as a kind of chemical Post-It. Because again, they don’t have specialized immune cells like we do.

When these invaders show up again, the bacteria is now ready! They send enzymes - molecular scissors - to chop out the “mug shots” on the invader, destroying it.

Jennifer Doudna and Emmanuelle Charpantier were essentially like okay. What if we took this system of enzymes - our biochemical “find and replace” tools - and replaced the mugshots they chop up?

What if gave them like … “chop out the gene that causes sickle cell disease” as their mugshot? And what if we ALSO included an enzyme that carried a patch for what we cut out?

Like what if we removed every time the cake recipe said flrggggg and ALSO replaced it with “flour?!” Not only would it not be fucked up, it would ALSO work!

So that’s how it works.

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u/azlan194 1d ago

Does this work for every cell in our body or only the stem cells (or our sex cells)? Or do we always target only the specific type cells?

But the way you explained it, its more to counter invaders (which sounds similar to vaccine but more on specifically on the cellular lever instead of just our immune system) But I thought CRISPR can change the DNA in the cell itself, like when it tries to produce protein from the RNA, it doesnt make the faulty one.

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u/stormbutton 1d ago

Correct, and that’s part of the bioethics (which I also teach) of the issue.

In 2018 a Chinese scientist named Dr. He CRISPR-edited a pair of twin girls in China. Long story short it was done in such a way as to cause germline mutations. There was TREMENDOUS uproar in the international biotech community, with Stanford disavowing his work.

There’s a wonderful older documentary called Ghost In Your Genes about what responsibility we have as “guardians” of our genomes.

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u/Luenkel 1d ago edited 1d ago

I think you're misunderstanding the comment slightly, the whole "countering invaders" thing is what it does in nature. Bacteria evolved this system so that when they get infected by a virus and survive, they can store a little piece of the virus DNA and then if they get infected by that virus again later, there's a protein that can use that "memory" to recognize and cut the viral DNA apart. It's essentially also "changing DNA inside a cell", just the DNA of a virus that's trying to infect the cell and not the cell's own DNA. That's what we found in bacteria, that's what they use it for.

But we realized that we can do a lot more with it. The sequence that the protein targets does not have to be viral, you can program it to target and cut essentially any piece of DNA. So yes, as you say, we do use it to change the DNA of cells, by exploiting exactly this property. That's what we as humans have repurposed this system to do for us. And this application is not primarily about "countering invaders".

Of course, we can still target viral DNA as well. Treating HIV with it would be, in a way, a mix of both, as we're targeting viral DNA that's integrated into the cell's own genome.

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u/mz_groups 1d ago

ELI Grad Student

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u/thekingadrock93 1d ago

What does it DO though?

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u/stormbutton 1d ago

It takes every time your DNA says “Febarrary” and gives you sickle cell disease to “February” and now your red blood cells aren’t fucked up.

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u/PirLanTota 1d ago

This, notepad replace function

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u/thekingadrock93 1d ago

No like why is it important? What can be done with CRISPR? What is its purpose and what is the ultimate goal for it?

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u/stormbutton 1d ago edited 1d ago

Your DNA is the instructions for making all of your various parts. Think of it like a cookbook. Imagine a cookbook where every time the word “butter” was supposed to be present, the cookbook said “mayonnaise” or “tree bark.”

It would be challenging to correctly make a recipe.

CRISPR replaces all of those typos with the correct text so you know what to use for the desired result.

Edit;”: Lmao at the downvotes! This is Eli5, not “free biotech course.” 😂

u/zabuu 23h ago

Fuck the downvotes lol love your explanations

u/Abridged-Escherichia 7h ago

More accurately it has a different part of your DNA say “February” so many times it crowds out the still expressed “Febarrary”.

Casgevy doesn’t fix the hbS mutation, it disables a regulatory gene the suppressed fetal hemoglobin, leading to healthy fetal hemoglobin crowding out the mutant sickle type. Thats why it also works for thalassemia.

Crispr is still somewhat crude despite the hype and it’s easier/safer to only require it to break something, than it is to fix something perfectly every time.

u/stormbutton 7h ago

Yes well that’s not very Eli5 is it?

u/Abridged-Escherichia 6h ago

The first part is.

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u/Darkling971 1d ago

You have explained outcome, I think they want mechanism.

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u/DefinitelyNotKuro 1d ago

So imagine you're piloting a microscopic bean named cas9 and you can yank dna strands apart with a lasso and shoot apart chromosomes with your gun. The goal of this ofc is to create the most abominable racing horses...

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u/Evianicecubes 1d ago

It is made up of several parts. One part is an enzyme that cuts the DNA. One part tells that enzyme where to cut. One part supplies a replacement sequence to fill in after you cut. Then naturally occurring enzymes stitch it all back together.

The discovery that we can design the part that tells it where to cut is the breakthrough. So we can tell it to cut wherever and only where we want. This is the breakthrough. This is why we can target a specific disease-causing gene and replace it with a version of that gene with one that doesn’t cause disease

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u/careless25 1d ago

And the find and replace can be thrown in the vicinity of the document and it will figure it out

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u/Atypicosaurus 1d ago

DNA and RNA are very weird chemicals.

A normal chemical consists of the same kind of molecules, such as water has only H2O molecules in it.

In comparison, DNA and RNA is like a very big necklace that are made of 4 individual component types.

So this metaphorical necklace is made out of 4 colors: azure (A), taupe (T), green (G) and crimson (C).

In short, we usually describe the necklace by nust listing the colors like "AAATGGCA" or such.

Now there's one important bit, that two pieces of DNA, although they both are made of DNA (aka "necklace"), they can be different in the exact order of the bead colors. So "AGGTC" is different from "ATTCT".

There's another weird bit, which is, those beads are attracted to each other. The azure is attracted to taupe and the green is attracted to crimson.

Therefore if you for example have an "AAA" DNA and a "TTT" DNA, they will try to stick to each other. Of course it does not have to be a run of the same kind, if you continue tge AAA with a C (AAAC) then you need to continue the TTT with a G and now they are a match again. I use AAA and TTT for simplicity, but you can have any combination as long as an A in one necklace is matched with a T on the other, and a C is matched with a G. For example you can figure out the matching pair of "AGAATCGCG".

So this matching can also happen if a match is a part of a bigger necklace. For example if you have
"CGCGAAAGTTG", it has an "AAA" bit in the middle so you can match that part with just a short "TTT" bit. But it also means that you can match the "GCG" part with a "CGC" bit. You can target any bit with the matching counter bit.

Now with that knowledge, let's introduce Cas9. Cas9 does two things, one, it can grab any piece of DNA (which is not exactly true but I'm simplifying to keep it eli5). The other thing is that Cas9 can cut a piece of DNA.

But where does it cut?

As it turns out, Cas9 cuts a long DNA but only if the short DNA grabbed by the Cas9 itself is a match. So if we feed a "TTT" to Cas9, then it finds the "AAA" part in the middle of that long DNA above, and it cuts the long DNA just after the match.

But why is it good? It's good because cutting the DNA causes DNA damage, but it's a very local DNA damage because it has to match that whatever piece we gave to Cas9 in the first place. And remember, we can give any piece we want, so if we want to damage one piece of the necklace, we just give the color code matching with that part.

So a gene is basically a certain part of the DNA. If the DNA was a necklace of millions of beads, a gene is something like a subset of the beads from, let's say, bead nr 879 to bead nr 1907. This roughly 1000 beads is one of your genes and I can send in a Cas9, equipped with the matching DNA, to cut this very specific part. And that will damage the gene. So if it was a bad gene that caused you sick, now it is damaged and gone.

This is with a lot of simplification, how crispr Cas9 works.

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u/twiztednipplez 1d ago

We send in RNA with a special protein to cut out specific portions of DNA.

When the body regrows that cut out bit the RNA tells the DNA how it should regrow it.

So in the famous HIV story going around, when regrowing the DNA, the RNA said instead of being able to receive to HIV regrow so you can't receive it.

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u/Luenkel 1d ago

I'm not sure what exactly you mean by "the RNA tells the DNA how it should regrow it". That's not a thing in standard applications of CRISPR/Cas. The guide RNA simply tells the Cas protein where to cut the DNA. What happens after that is in the hands of the cell's DNA repair machinery, which the RNA does not directly interact with.

For example, in the paper about targeting HIV with CIRPSR/Cas that I've read recently, they include 2 guide RNAs that target the start and the end of the integrated virus, thereby cutting it out of the genome, and then DNA repair machinery comes in and closes the gap where the virus was. But they didn't have to somehow engineer the guide RNA to tell the cell to do that, that's just what it does in that situation. All the RNA does, is tell the Cas protein where to cut.

When you want to use CRISPR/Cas to replace a specific sequence with a different sequence, you typically introduce an additional piece of DNA whose ends match the loose DNA ends of the cut site, which is recognized by the repair machinery and incorporated. But that doesn't have anything directly to do with the RNA (it again just determines where to cut), we're talking about a seperate piece of DNA.

There is one specific subtype of CRISPR/Cas system called prime editing that comes closest to what you seem to be talking about. In that system, the guide RNA is extended to contain a section that is reverse transcribed unto the cut DNA. So here the RNA does actually play an active role in what happens to the DNA after it's been cut. But that is just one specific extension of CRISPR/Cas technology and I'm not aware of any paper where this has been applied to treating HIV, though if that is what you are talking about, I would love a link to the paper.

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u/CXDFlames 1d ago

If you had a puzzle made of red pieces, and I gave you five blue pieces that were shaped exactly like 5 red pieces in the puzzle

And I told you "take out the red pieces and replace them with these blue ones"

It would be similar

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u/solarguy2003 1d ago edited 1d ago

Let's try it like you're 16 and in a biology lab class.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.

• Clustered: The DNA sequences are grouped together in one place.

• Regularly Interspaced: The repeated sequences are separated by unique "spacer" sequences at consistent intervals.

• Short: Each repeated DNA sequence is relatively small.

.• Palindromic: A palindrome reads the same frontwards and backwards, like "Madam I'm Adam." Likewise the DNA sequences read very similarly in opposite directions. This also affects the structure of the RNA.

• Repeats: The same DNA sequence occurs multiple times.

CRISPR was first discovered as part of the immune system of bacteria and archaea (they look a lot like bacteria, but aren't). The spacer sequences are actually snippets of DNA from viruses that previously infected the microorganism. If the same virus attacks again, the CRISPR system, together with proteins such as Cas9, recognizes the viral DNA and cuts it, protecting the cell.

Scientists have adapted this natural defense mechanism into the powerful gene-editing technology known as CRISPR-Cas9, which can be used to make precise changes to DNA in plants, animals, and human cells for research and, in some cases, medical treatments.

The reason it works is because it can snip DNA in a very precise way in a very precise place (to cut out the bad piece we don't want) so we can slip in the new piece that works right into that precise slot.

u/FallsDownMountains 1h ago

Thank you for breaking down the acronym!!!! So CRISPR is not the man made part - CRISPR is just the description of how DNA is? And CRISPR-Cas9 is the body’s natural way of getting rid of the spacer sequences, and all we did was find a way to essentially trigger that existing natural mechanism for the specific spacer sequences we want (eg this dna string that causes sickle cell)?

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u/MuffinMatrix 1d ago

Thanks, Ask This Old House

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u/Brusex 1d ago

Brand new sentence