How Gene Editing Is Curing Disease
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- Опубликовано: 21 авг 2020
- Watch this video ad-free on Nebula: nebula.tv/videos/real-science...
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Patreon: / realscience
Twitter: / stephaniesamma
Instagram: / stephaniesammann
Credits:
Writer/Narrator/Editor: Stephanie Sammann
Editor: Dylan Hennessy (www.behance.net/dylanhennessy1)
Illustrator/Animator: Kirtan Patel (kpatart.com/illustrations)
Sound: Graham Haerther (haerther.net)
Thumbnail: Simon Buckmaster ( / forgottentowel )
Producer: Brian McManus ( / realengineering )
References:
[1] www.cdc.gov/ncbddd/sicklecell....
[2] www.npr.org/sections/health-s...
[3] www.quantamagazine.org/crispr...
[4] sitn.hms.harvard.edu/flash/201...
[5] ir.crisprtx.com/static-files/d...
[6]www.nature.com/articles/d4158...
[7]investors.vrtx.com/news-relea...
[8] www.texaschildrens.org/health....
[9] www.vox.com/2018/7/23/1759486... 
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I am one of the people that Crispr cured. I was cured at children's hospital of Philadelphia after 28 years of Sickle cell SS! I now have more energy than ever before, my eyes aren't tinted yellow anymore, I can go out in the cold and not sickle. I feel like they just gave me a whole new life honestly. I want this to help everyone who suffers from sickle cell.
damm hope they figure it out for beta thalassemia too. That's what I have. they are not the same but similar.
Hiiii, please tell me how you were able to have access to this ?!?? I want to be cured as well
@TRINIGIRLHANNAH speak to your doctor about any stem cell trials. Mine was able to link me to children's hospital of Philadelphia where they cured me using my own stem cells
@@C0smiChr1s can you plz tell me how much money required for treatment ??
@silverscreen8119 because it was a trial I didn't pay anything but they are saying it costs them millions of dollars
Dylan. I know that was you yeah little bollix.
Ahah “Real science is the best RUclips channel”
Been wondering this for a while, what exactly is your pfp image
@Advait Shukla History is important because every invention and technological marvel exists because of certain specifications and limitations, which depend on the situation. Something like explaining how a fighter has been designed requires context of what limitations there were and what purpose it was meant to fill. It's also just plain interesting. I would be really interested in the topic you suggested though.
When will we get a Collab? XD
(Btw I know you guys own both channels)
@@danielmartin9057
I believe it's the profile of an involute gear tooth.
The horrible experience of watching a loved one during a sickle cell crisis being utterly helpless to stop the pain that's beyond agony I'M SO GLAD TO HEAR THERE'S HOPE FOR US IN THE IMMEDIATE FUTURE
Thanks for the episode, I love how DNA technology is advancing so quickly.
Yeah!
Me too
'so quickly' hahaha
You could almost say............... It's evolving
It's good but it WILL be bad. Governments and militaries will get more involved than they probably are
As someone who just had an organ out due to a genetic illness, I feel both hopeful and cheated by time after watching this.
i pay close attention to the gene edit field because i have Ehlers-Danlos syndrome(hyper mobile type) I hope one day to be cured of it through crisper and pray many more will too
@@Grimwalkerx Hearing of that syndrome for the first time....can you do a split naturally? :D
@@scheichajev lol im not surprised it pretty rare to find a doctor that's heard of it, even less for others. and yes i can. for me im double jointed in almost every joint, this means i dislocate or sub locate bones often to the point im disabled. even with that tho i can thankfully still walk with the help of a cane outdoors and without one at home, if all the covid 19 crap didn't start i would have been doing physical therapy but i didn't want to risk it bc im at higher risk , mostly bc eds puts a strain on your body by being in a state of repair 24/7 bc the body's always tearing the cologne in the body for me, it in the chest the most.
@@scheichajev if you have any questions about eds im happy to try to answer :)
Just wait 5 years. Everyone says it's a year away but it's probably five years away if you're being conservative
This is going to change humanity forever once mastered.
@eblman Same here
Gene editing might just be the most needlessly feared and underused technology of today.
@Sandcastle • That's a bit of an exaggeration. There's only around 1.5 billion people born per decade, and the vast majority of them wouldn't benefit from our current capabilities. For that matter, we don't have the industry of scale to help everyone--even for clear-cut cases like the sickle cell treatment, it's way too complicated and expensive to provide to everyone who needs it.
I work as a PhD in a gene editting lab using CRISPR technologies to study cancer. It is not needlessly feared. There are some things that we should resolve and be concerned before we use genome editting widely. The number of different types of proteins in body is estibated between 80.000 to 400.000. Basicly, we dont even know how many proteins there in the body. We know one function for maybe quarter of those proteins. The remaining 75% has unknown function. Add the fact that each protein can have several different functions and dozens of interactions with different proteins to for more complex structures to accomplish more complex tasks. We dont know in which type of cells, which of those 80 to 400k proteins are activated. We cant even make a good categorization of subtype of different cells. Thus, we can not even imagine the all consequences of each editing in molecular scale. Yet alone cell scale, tissue scale, organ scale, system scale, organism scale. Because we dont know enough about the rules of the cell yet. For example maybe the transcription factor they edited out is a currently unknown tumor suppressor. And editing that out will result in increased tumorigenesis in these edited cells. And that person will maybe develop cancer due to that editting. We currently dont know for sure, since we dont know enough about the cell yet. That is why genetic engineering has a lot of risks to be assessed before it is used worldwide. By the way there are also several other problems else than that. One last I can mention before it gets too long is the specificity of CRISPR. Yes it is very precise editing compared to older editting tools. Because guide RNA binds to DNA very precisly based on its sequence. Cas9 cuts at the precise location based on guide. However, in real life things doesn't work perfectly. Even if their sequences differ with 1 nucleotide, there is a small chance the guide RNA bind to off target locations with 1 or 2 mismatch. Which means it might binds to off target locations despite not being perfectly complementary to that location. Cas protein can cut at wrong location. Things doesnt work perfectly in cells. But when you apply a therapy to a person, you dont have the luxury of saying of there was a 5% chance of off target cutting and now you have increased chance of cancer. There are rules regulating what you can put into the medical market as a "therapy".
I hope it would be explanatory :) Have a nice day
@@aguven thanks for all that information. I learned a lot in reading what you said, in addition to the video.
@@aguven
- I agree that there are risks associated with gene therapy. My point however is that people fear it too much and for the wrong reasons. And not only when used in humans but especially in agriculture. People suddenly fear a plant when it's edited with CRISPR but will happily consume plants that have been heavily modified by chemicals and radiation over decades. Because somehow that's more "natural".
- now to the specificity point. Studies in recent years on human embryos seem not to detect an appreciable rate of off-target effects. Modern tools for gRNA design greatly reduce the risk of them.
A miniscule chance will still happen if we edit billions of cells however. But as a researcher on cancer you should be well aware that one random mutation is unlikely to produce cancer.
So, in a child with a very well understood genetic disease (let's say Duchenne muscular dystrophy) it is very likely that we can simply cure his up to now uncurable disease with a miniscule chance of raising his risk for something else.
@@Pyriphlegeton I agree
Real Science and Real Engineering have done it again. Thank you for this amazing content! Your channels are my very favorites.
Thank you for making concepts look simpler, the annotations and graphics are highly intuitive. Please continue to make videos on medical sciences, keeps me motivated to learn more.
I needed to rewatch this 3 or 4 times to understand it.
It was worth it
Love this channel ❤️ Get to learn something new from every episode, Keep up the great work guys 🤗
This is unbelievably amazing subject which just makes me so intrigued that I can't express!!!
"Sorry Brian, it's true" 5:32 Top left
why
This was really interesting! I wrote a 5000 word dissertation on gene therapy for pancreatic cancer this year for my EPQ (UK qualification for 16-17 year olds) so always love to hear more about this topic :)
May I know if your manuscript is available online for us to read or download. Thanks in advance!
Yeng Sabio I really ought to share it online. If you want I would be more than pleased to send it to you!
Yeng Sabio ok here is the link. Let me know if it works? docs.google.com/document/d/19Ht05jCHPE8G9FLO7JQXpD0o5z-_EBn5kcgbDQlr488/edit
Will Green really interesting read. Have you considered publishing it even just on something like medium?
@@willgreen8310 That's a solid report! Is this standard in the UK education system? Based on what I found on Wikipedia, an EPQ counts as half an A level course (which take two years), so it counts as a full year course?
That sounds similar to a program my highschool did, the Cambridge Capstone Program. But our papers weren't nearly as scientifically rigorous!
If you want to see some more cool science, check out the Institute for Protein Design / Rosetta Program -- I did a semester of work there as an undergrad, and I'm super excited about where that work will go in the future
This is the best description of CRISPR Cas 9 I've ever heard.
but are you a scientist?
@@userou-ig1ze Yeah, actually I am. Not a molecular biologist, but I am a scientist in an allied field.
@@incognitotorpedo42 but what allied field?
@@userou-ig1ze For what it's worth, I got my bachelors in biochem/molecular biology, and this is also the best description of CRISPR/Cas9 I've ever heard.
As far as I recall, we had one day on it in an introductory biology course; everything else has been from informal education (such as this video! =D)
Wow, extraordinary work on the video! Subscribed!
This channel is freaking awesome! Totally worth the wait between uploads! Just keep it up as it is, Cheers! ;)
The future is bright if we can (responsibly) roll this out to the masses!
Ok, so finally after a month i got to see a video from real science.
This is the best science channel I have ever seen + your voice is soo good.
I guess i will get a subscription for curiosity stream and get a subscription for nebula.
Thanks you very much for your work on this video it is such an interresting subject.
I espacialy enjoyed the CRISPR explanation.
Gene Editing sounds so crazy futuristic, man I love science so cool!
Same
Your videos are getting better and better, young lady!
Real engineering is the best youtube channel.
No, No its real science 🤣
I guess real things are just better ;)
No, No both of them are the best
yea, ikr 🤣🤣🤣😂🤣🤣😂☺️☺️🤣🤣😉🙃🤣☺️🤣🙂🤣☺️🙃😂🤣🤣🙂😂🤣🤣 (insert more emojis here)
Ur so funny 😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂😂
Oh you all have a got a sense of humor! 🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣🤣
i was liturally searching for crispr news because i find it so interesting.
But i couldnt really find much.
I am very happy that youtube showed me this video, its amazing :)
Yes yes yes yes yes yes to the nebula platform! I bow to you in deepest gratitude! I have been pressing the idea of an alternative to facebook and RUclips since a couple years after each platform came out. But alas I am not intelligent enough to physically create either. I have been awaiting this for so long! Thank you to every single one of you brilliant contributors that pulled that off! Great video content and clear concise explanations. I'm sure I'm late as hell finding all of this but its brand new amd great to me! Thanks!
I've been hearing about CRISPR on RUclips, but this is the first video I've watched that clearly explains the history, and most importantly, how it actually works!
Before this, CRISPR was some magic tool, but now I realize its mechanics are pretty simple
What a GREAT video! I mean, it´s just what we are used to see both over here and Real Engineering. Thank you!
The future top search term for CRISPR: "How can CRISPR increase the size of my-"
😂😂😂😂 got you!!
Muscles of course .
Brain
Second would be regrow my hair
We all know what you're talking about....
Lifespan, duh
My cousin has severe sickle cell disease, he has to go to the hospital multiple times per year, he has to get his blood replaced each time. Bone marrow transplants are too risky so he won’t get one.
Awesome episode, I love that CRISPR is actually translating to helping people in a clinical setting, which takes a lot of time in all of science from research or discovery to practical application(s), I have one small correction - gene therapy and genome editing at the level of somatic cells have existed long before widespread CRISPR-cas9 based protocols. Around 2011-2013 we got the nice CRISPR instruction manual paper(s) culminating in the ran et al., 2013 paper labs use for mammal applications of CRISPR. [1]
I'm focusing on adult somatic cell mods here, which have been done in research using ZFN and TALENS, although CRISPR-based systems may promise greater specificity with less off-target effects, the main benefits IMO are the low cost and ease of access with easy programmability leading to potentially greater success rate with a low cost, i.e. widespread use and availability.
It's like saying before 1960 no cars could travel at 140 mph, there were such cars, but now CRISPR can be had for a few 100 USD instead of thousands or more for a single test of gene therapy - overall treatment costs are obviously more than this, but just considering the reagents and cas proteins required.
It's great that such a powerful tech is now available but I hope we can use it responsibly and recognize that CRISPR isn't a magical machine that eliminates all off-target effects. Any secondary off-target effects that hit other essential genes with a similar sequence should be thoroughly investigated for each given single gene disorder/single gene edit, and consider the effect of possibly removing code from one of the most interconnected and complex systems we know of (i.e. mammal/eukaryotic/human genome).
However when used in bacteria, I am very comfortable using CRISPR because I'm not that concerned with the quality of life of my cell culture as long as they make my recombinant target and serve their purpose, but for humans even for a small and localized alteration like targeting a single gene in blood, liver tissue etc it could have profound consequences that could take a while to truly see even if your model is very sophisticated and has shown where possible aberrant cuts could be made; we can never be 100% sure what the effect(s) would be - all genes control other genes, some indirectly (like getting a cut activates the immune system, but the cut itself doesn't produce white blood cells), and many direct, like how about 1500 regulatory genes exist in humans to regulate the 20 000 or so other genes [2], so these controller genes (ie transcription factors, TFs), are used to activate a gene, and 20 some TFs must come together and regulate the activity of any single gene, so messing with one TF doesn't affect only one gene, it could a dozen genes, although there is some redundancy built in.
[1] Ran, F., Hsu, P., Wright, J. et al. Genome engineering using the CRISPR-Cas9 system. Nat Protoc 8, 2281-2308 (2013). doi.org/10.1038/nprot.2013.143
[2] Ignatieva EV, Levitsky VG, Kolchanov NA. Human Genes Encoding Transcription Factors and Chromatin-Modifying Proteins Have Low Levels of Promoter Polymorphism: A Study of 1000 Genomes Project Data. Int J Genomics. 2015;2015:260159. doi:10.1155/2015/260159
Really good explanation of CRISPR, thanks!
A friend of mine died of sickle cell anemia 2 weeks ago on the 15th of July 2022, which is 2 years after this video. He was only 23 years old. I feel impotent rage against this technology for not helping him, and yet I dream of hope.
Thank you for your detailed explanation of CRSPR
So informative! My new fav channel
Awesome article thanks.
As a sickle cell patient, this was a little dose of hope.
Yes! Can we please get more videos on this topic. A video on epigenetics would be greatly anticipated.
media needs to cover this & more money needs to be given to this type of research
Great video! Super interesting and it’s really cool seeing the numerous applications for crispr!
Just a trivial, technical question about the sickle cell treatment... Aren’t BCL genes related to tumor suppression? Wouldn’t we expect an increased risk of leukemia in patients transplanted with cells containing this BCL11A deletion? Is there a reason why this isn’t a significant concern for these treatments?
Thank you for the information very informative
This video for me finally completed what CRISPR really is. Love your videos. There is always new to learn here❤️😎.
I love how CRISPR went from
Will cure
TO
Is curing.
Hopefully we will get in vivo gene editing soon
Great episode, have only exploded epigentics a little but now I'm definitely motivated 😊
Very nicely done news piece.
Very well explained!!
5:27 Shots fired.
5:27 That Shade..!!
Love it 😆
SCA was used to explain genetics in my HS Biology class, and it was initially caused as an adaptation against Malaria. It’s good that we know how to reverse a backfiring adaptation.
I had a professor who worked on a therapeutic for sickle cell disease for years. He realized our gene editing revolution would make his therapeutic obsolete. I don't think he cares, I think he's just happy nobody will need to suffer from sickle cell disease some day soon
Good explanation, with good visual aid :)
Very encouraging results albeit not without issues with bone marrow rejection.I’m not sure what I find more amazing, the fact that we have figured out what the palindromic sequences function is, or that bacteria have evolved to combat virus infection by this intricate process. Is this treatment based on EDIT-301 developed by Editas ?
I think this video did a good job of advocating the promise of CRISPR while acknowledging the drawbacks and unknowns that warrant rigorous caution. Good stuff!
Whether through CRISPR or other means, I hope we can continue to find ways to help the sick
Wonderfully explained.
Dang!!!!! no mention of credit to Dr. Mojica. CRISPR mechanism and name was originally discovered back in the 90s by a particularly humble microbiologist, Francisco Mojica, Professor at the University of Alicante. He had the insight.
thx u Generation x for ur work
love your work keep it uppp
Real engineering got roasted 😂
i’m hype for this channel
Insightful and enlightening 💡
Deleting one line of code may solve one problem but may create several bugs - frustrated developer 😬
Or it could solve several problems with only a few, if any, bugs. We won't know until further research is done.
So speculating the potential dangers won't get us anywhere unless we test it rigorously.
Yup! We can't go making edits until we understand the whole system (which, we don't. Yet.)
But in cases like these, the benefits far outweigh the risks, and we have a pretty good idea what effect an increase in hemoglobin will have.
@@tomc.5704 Exactly
@@tomc.5704 This goes beyond sickle cell disease. People who may have a cancer gene may be able to get it replaced with something that could boost the immune systems response to cancer. Due to the fact it won't cure them because of environmental factors but it would certainly decrease the number of incidents of cancer globally boost survival against Malaria if not a complete immunity. I'm excited for this only problem about this is that there are very very strict guidelines and only for clinical trial usage only which sucks
@@lukecarmody2446 there isn't a cancer gene, cancer is a genetic defect. The increased risk of getting cancer can be inherited however.
Thank you and nice video for understanding
nice work, keep it up
Very informative and magical CRISPR technique.
Th upload speed and quaility of theses videos are exponential
Since I haven't seen him mentioned here, I wanna plug The Thought Emporium.
He does a lot of CRISPR stuff on RUclips and even produced a gene therapy to cure his extreme lactose intolerance. Amazing science channel!
Ps great work on the vid!
Oh that was awesome! Thanks for that video
This is great!
Well Done, Thank You!
This is a great introduction to this topic!
And hooo boy, y'all just wait for what we can do in the future. Chemotherapy and a bone marrow transfer is cutting edge today, but will be seen a crude technique.
Right now, we don't fully understand how the body works. We don't know what enzyme or signalling protein would grant us access into the cell to perform these edits directly. But we know a hundred times more than we did thirty years ago, and we have labs working on these questions, and we have labs learning how to create biological nano machines (i.e., proteins).
I kid you not, we have a path towards curing every disease. Every. single. one. It's a long and difficult path, but we can finally see it. We probably won't get their in our lifetimes, but we will see progress. (And if we get quantum computers to a useful point, we will see a LOT of progress. The entire Rosetta@Home program would be trivialized by a quantum computer.)
This eleviates my mood despite the stuff happening in 2020.👍
Kurzgesagt fans: Hey, I’ve seen this one!
RealScience: What do you mean you’ve seen this? It’s brand new!
Yeah!
this video feels clearer, dunno if it's just me forgetting the kurzgesagt video or not
@@naufalap nah it is. Kurzgesagt only mentioned it for about 1-3 minutes. This is an entire video
She is 4 years too late
@@thealpaca18 This story about CRISPR being used to successfully treat sickle cell was first published in November 2019.
Sure, we know about CRISPR before this, but now we're seeing proof of concept!
Real Science > Real Engineering
Jk...😅😅😅
They are both Great
doing an undergrad in genetics atm this is all so exciting of what's possible
Saw this on a Stargate SG-1 episode after Teal'c took a bug bite from a very big bug.
Man. That is complicated stuff. Amazing they can figure it out
Amazing!
I like how this video is done. Often when someone creates a brother/sister channel or hire someone to take some work off of his shoulders it suffers because the new person brings too much of his/her personality or too different a personality than I've gotten used to. Here it's very similar to real engineering. I like that.
Can CRISPR fix my relationship?
Theoretically yes, as long as your relationship problems are due to genetics, like your gf being unsatisfied with your dick size
of course. Just insert guide RNA to replace your girlfriend
@@thonktank1239 Hahahahaha
No but it can help you take Moscow
No, unfortunately CRISPR cannot create another organism
WOW!! This is amazing content. 10:23 the computer chip from Lain is real.....
Several times I have followed links to the Curiosity Stream website and gone through the sign up procedure only to find that there is no options for PayPal, Amazon or Google Pay. I won't use my bank details/card to sign up for one service which isn't even in my own country. Please suggest they look into more ways to pay.
Absolutely amazing science.
This is great - I didn't know that CRISPR had actual medical use already. The last I heard of it, I thought they'd run into the problem that the alterations wouldn't just be targeted in the right spot, but caused a similar change in many other locations as well. Maybe they overcame that problem?
PAM Sequences - A protospacer adjacent motif is a 2-6-base pair DNA sequence immediately following the DNA sequence targeted by the Cas9 nuclease in the CRISPR bacterial adaptive immune system.
Incase, anyone was wondering like i was!
I always forget about it’s meaning, thank you
Cool, crispr can also be used to regulate gene expression, not necessarily cut and paste stuff. Fascinating stuff.
👏to advance in gene mapping and molecular biology;
btw, one suggestion
please popularise the usage "disorder" (# "disease") in relation to SCA 🙏
absolutely amazing
I have been reading about this CRISPR technology for years and I expected we understand and can make good things more from it then just what it can offer to us now. much more.
One question how much to get this done?
Great video.
Can you do a video on "cerebral palsey". And more about neurology...
Please, to make life easier for Nebula subscribers, could you add a direct link to the nebula version of videos in the description of future videos?
Thanks !
Could it be that you Switched up Tom With Joe scott in the curiositystream/nebula commercial? Best regards
5:28
Shots fired!
would this work on sars-covid19 Rna?
"you can never feel my pain". RIP Prodigy
Yesterday ,I read about the history Of CRISPR .
This can be used to make super soldiers...... I love it. Sign me up!
Not an expert but best explanation of crisper ive seen
I see potential of this technique in baldness therapy.
@Fareed Waseem excellent analysis. Best luck to you 👍🏻
How did they reduce the off target effects of crisper in this treatment ? (Crisper has hughe off target effect rates )
*Feedback for the channel:*
One part about ~4:26 wasn't clear to me.
I would've assumed the anti-virus DNA of the cell attacks the DNA virus itself.
But what the animation shows is that the virus must have already infected the cell's chromosome(s) with its DNA, and now gets removed again (or neutralised, at least).
Is that correct?
Does the cell DNA neutralise the virus DNA *after* it's become imbedded in the cell's DNA, instead of intercepting it before it embeds itself into the cell's DNA?