The First CRISPR Gene Therapy Is Here

CRISPR is a powerful gene editing tool, but its uses have been purely scientific until now. In 2023, US and UK drug regulators including the FDA approved Casgevy, a CRISPR/Cas9-based therapy for sickle cell disease and beta-thalassemia. The FDA also approved a similar treatment, Lyfgenia. Here's how they work.

Hosted by: Reid Reimers (he/him)
----------
----------
Huge thanks go to the following Patreon supporters for helping us keep SciShow free for everyone forever: Adam Brainard, Alex Hackman, Ash, Benjamin Carleski, Bryan Cloer, charles george, Chris Mackey, Chris Peters, Christoph Schwanke, Christopher R Boucher, DrakoEsper, Eric Jensen, Friso, Garrett Galloway, Harrison Mills, J. Copen, Jaap Westera, Jason A Saslow, Jeffrey Mckishen, Jeremy Mattern, Kenny Wilson, Kevin Bealer, Kevin Knupp, Lyndsay Brown, Matt Curls, Michelle Dove, Piya Shedden, Rizwan Kassim, Sam Lutfi
----------
Looking for SciShow elsewhere on the internet?

#SciShow #science #education #learning #complexly
----------
Sources:

Images:

[MitigateOurRuin]

A few months ago, my medical school invited the first person treated for sickle cell disease with this technology to lecture about their experience, and I had the opportunity to meet them. Indeed, we live in exciting times!

[aronbakker]

In the Netherlands a small group of patients with hereditary angioedema has been treated with this technique. The results are astonishing. It is able to give people back their lives. Of course this disease is a relatively easy fix but it is really promising.

[mystuff]

„The floodgates are open“ is the most important sentence here. This is going to rapidly produce at least better treatment for a plethora of diseases if not a cure.

I would be very interested to know how the study explains certain people not seeing improvement. I don’t really understand how that’s even possible with this approach 😢

[isuperninja]

I have actually worked on these clinical trials in the lab, it was pretty amazing to see how fast the condition of these patients‘ blood cells improved

[KuraiKaNinja]

before i became chronically ill and disabled i was studying to be a geneticist to do exactly this. I first heard of CRISPR when i was in high school and couldnt wait for it to be used to treat conditions we've had very limited ways of treating. while i havent been able to contribute to research myself im still super hyped to hear about these advancements!
i look forward to hearing more about this in the future 🥰

[kthfox]

"thalassemia?" i asked myself. "why that seems to translate to 'sea blooded'. what the heck is that supposed to mean?" it's because it was first noticed among people around the Mediterranean sea. ah etymology.

[BlareWolfgang]

I had a bone marrow transplant 15 years ago for an autoimmune disease called aplastic anemia. Thankfully I have a lot of siblings and one of them was a perfect match so I didn’t have to wait on the donor list. It’s cool that now people are getting a chance at similar results without needing a donor because you are the donor. Great stuff 👍

[SoulDragonWithFlow]

I'm hoping that one day my damaged spinal cord (C2 SCI) might be repairable or regenerable so I can move again. Maybe CRISPR holds the key, maybe something else, but I'm not holding my breath. Always good to hear when some kind of STEM advancement leaves just a few less people suffering in the world.

[Dabuddah]

To be fair, these things tend to get cheaper as time goes on.*

*So long as the development process is not held by a singular company.

[nobleeverett]

It's wild that when he says "If I had a nickel for every gene editing treatment for sickle cell approved in December 2023... Well, You know how this one goes." The assumed conclusion is the Phineas and Ferb reference "I'd have two nickels, which isn't a lot but it's weird that it happened twice" instead of the original idiom "I'd be rich". The Phineas and Ferb joke has replaced the (maybe, probably, in it's modern incarnation) Mark Twain stock phrase and now "If I had a nickel for every time" implies by default it's happened 2 times not many

[chiedzawithds]

Seeing a SciShow episode on CRISPR is the reason is wanted to study genetics. And now nearly done with my degree. 😊

[Tuna-jwxj]

This is what technology should be used for. This is amazing.

[ThomasShaneHynes]

As someone living with terminal ALS this gives me hope that CRISPR can evolve enough to cure this horrible illness too.

[jamesdoughty]

Maybe we should thank Jennifer Doudna for inventing CRISPR, she got the Nobel Prize in chemestry and the French lady as the co-inventor.

[MicrowaveOvenVideo]

I am a pre-med student in Nashville and I work in the hospital (Sarah Canon: Tristar Centennial Hospital) where she was treated. Absolutely insane being able to work with the people who took these first steps!

[Christopher_S]

The people taking part in this experiment are legends and heroes to me. I've been waiting and waiting for this to become available for myself.

I've got an auto-immune disease (with no cure) called Ankylosing Spondylitis. I'm one of the lucky few where treatment is difficult to get to work and my disease is significantly more aggressive and advanced than what others have.

CRISPR will theoretically fix me, but until now it's never been allowed to be used in humans. If this works for these patients, then there's hope researchers will work on a treatment for me to finally cure me.

I can't wait at all. CRISPR is the only reason I haven't taken my wheelchair to the edge of a cliff and forgot the brakes.

Speedy, safe and problem free recovery to everyone involved!

[Offutticus]

I have a genetic disorder called Ehlers-Danlos Syndrome that effects the collagen of every cell, with varieties referring to which cells are the most effected. One variety of it is vascular and causes patients to die of ruptured aortas. Another variety, mine, causes loose joints, chronic pain, and repeated dislocations. I'd do (almost) anything to make the pain go away. Not sure there is anything to be done about the already damaged joints, but body-wide working fascia? Sign me up!

[EmilyJelassi]

I really, Really hope they can insert the missing Proteins in My DNA soon!! Am missing 2 out of the 4 Proteins in My DNA that makeupmyautoimmune system; I'm the only person in the world who's been found to be missing more than one. .. it doesn't even have a name yet!
But im so happy that CRISPER is finally in the hands of doctors who can use it to help people!!! 🙌 😊🎉

[astralb.]

Crispr feels like my kid growing up... good job buddy!

[Skenjin]

After this helps people with deadly blood disorders, I hope it can be extended to people with autoimmune disorders.