We Love Science

Ep 47: The CRISPR Cure for Sickle Cell Disease

April 07, 2024 Shekerah Primus & Fatu Badiane-Markey Season 3 Episode 15
Ep 47: The CRISPR Cure for Sickle Cell Disease
We Love Science
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We Love Science
Ep 47: The CRISPR Cure for Sickle Cell Disease
Apr 07, 2024 Season 3 Episode 15
Shekerah Primus & Fatu Badiane-Markey

Send us a Text Message.

In this fourth installment of the CRISPR Chronicles, show hosts Fatu and Shekerah give  a review of sickle cell anemia, the cause and symptoms of the disease, and discuss how the CRISPR gene therapy treatment works.

Tune in to learn more about:

  • The genetic basis of Sickle Cell Disease
  • What Amazon delivery vans have in common with your Red Blood Cells
  • The effect of mutant hemoglobin on Red Blood Cells
  • The variety and severity of symptoms that SCD patients experience
  • The disease burden of SCD throughout the world
  • The clever strategy used to treat the disease with CRISPR gene therapy
  • The results of the CRISPR Therapeutics and Vertex Pharmaceuticals gene therapy clinical trials

    For more information on this topic, visit our website: welovesciencepodcast.com

Hear directly from Sickle Cell Disease patients who received the treatment during the clinical trial: Victoria Gray and Jimi Olaghere

Catch up on the previous CRISPR episodes:

 

Reach out to Fatu:
www.linkedin.com/in/fatubm
Twitter: @thee_fatu_b
and LoveSciencePodcast@gmail.com

Reach out to Shekerah:
www.linkedin.com/in/shekerah-primus
and LoveSciencePodcast@gmail.com


Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoor
Music from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic

Show Notes Transcript

Send us a Text Message.

In this fourth installment of the CRISPR Chronicles, show hosts Fatu and Shekerah give  a review of sickle cell anemia, the cause and symptoms of the disease, and discuss how the CRISPR gene therapy treatment works.

Tune in to learn more about:

  • The genetic basis of Sickle Cell Disease
  • What Amazon delivery vans have in common with your Red Blood Cells
  • The effect of mutant hemoglobin on Red Blood Cells
  • The variety and severity of symptoms that SCD patients experience
  • The disease burden of SCD throughout the world
  • The clever strategy used to treat the disease with CRISPR gene therapy
  • The results of the CRISPR Therapeutics and Vertex Pharmaceuticals gene therapy clinical trials

    For more information on this topic, visit our website: welovesciencepodcast.com

Hear directly from Sickle Cell Disease patients who received the treatment during the clinical trial: Victoria Gray and Jimi Olaghere

Catch up on the previous CRISPR episodes:

 

Reach out to Fatu:
www.linkedin.com/in/fatubm
Twitter: @thee_fatu_b
and LoveSciencePodcast@gmail.com

Reach out to Shekerah:
www.linkedin.com/in/shekerah-primus
and LoveSciencePodcast@gmail.com


Music from Pixabay: Future Artificial Intelligence Technology 130 by TimMoor
Music from https://freemusicarchive.org/music/Scott_Holmes: Hotshot by ScottHolmesMusic

Shekerah Primus  0:15  
What can you do with your love of science we'll tell you

hello, everyone,

welcome back to your favorite podcast. We love science. We are your hosts. I'm Shekerah,

Fatu Badiane-Markey  0:50  
and I'm Fatu and today we have another crispy episode for you guys

Shekerah Primus  1:00  
Yes we do. We're back with another all CRISPR episode. And this time it's all about Sickle Cell Disease. But before we get started how are you doing today Fatu?

Fatu Badiane-Markey  1:12  
Yeah, I'm doing pretty good. You know, it's been a little bit of a lazy day coming off the weekend. Not not too bad. I got a lot of sleep. On Saturday. I slept like almost the whole day. My body was just like in reboot mode. I don't know what it was. So today, I'm feeling very refreshed.

How bout you  Shekerah? 

Shekerah Primus  1:38  
reboot mode what are you rebooting from?

Fatu Badiane-Markey  1:40  
girl I don't know.

Shekerah Primus  1:45  
All right, the body gets what the body needs.

Fatu Badiane-Markey  1:47  
I think I just needed it Yeah, yeah,

Shekerah Primus  1:50  
I'm all for lots of sleep. So that's okay. I'm doing pretty good too. Actually. It's another spring like very nice day here. It's still a little chilly though. Because it's like low 50s here, but at least it's not raining. We had some pretty bad storms at the beginning of the week. So okay, yeah, at least it's it's not raining. It's nice today and you know the trees are flowering and soon they'll be green again. So yes, spring is always a nice time of year like a new beginning

Fatu Badiane-Markey  2:24  
Yeah I love spring. It is it is a really nice time of year. It's really nice time of year. Yeah.

Shekerah Primus  2:31  
And it's also like this is just the beginning of April right but it's already like getting dark. Like not till eight o'clock. So I'm like oh my gosh, I feel like It's staying bright later than normal for beginning of April. happy about that. It's nice to have a little bit more daylight.

Fatu Badiane-Markey  2:50  
Yeah. All right. Always Always. 

Shekerah Primus  2:53  
Yep So today's episode is all about sickle cell disease. So what is it? What are the symptoms, the treatments and the CRISPR cure? Right. So CRISPR gene therapy treatment, I guess is what I should say. which is effectively a cure based on the results from patients who received this treatment. Right. So I think it's fair to say the CRISPR cure.

Fatu Badiane-Markey  3:20  
Yeah, I agree with that. You know, I'm just thinking back to when we were younger, you know, probably like middle school high school biology, we're learning all about sickle cell disease, you know, and back then, I don't even think people had any ideas that a cure would be on the horizon, you know, maybe like 15, 20 years later. So this is such an incredible story, and I'm really excited about this episode. So Shekerah, let's start with the basics. What is sickle cell disease?

Shekerah Primus  3:51  
Yeah, I'm gonna teach y'all all about sickle cell disease today.

Fatu Badiane-Markey  3:55  
We're ready to learn. Go ahead girl.

Shekerah Primus  3:57  
So Sickle Cell Disease is a genetic disorder that affects red blood cells. So it's caused by a mutant gene that you inherit from your parents. And as we know, red blood cells are the cells that carry oxygen from your lungs. So when you breathe in, right, the cells act as the transport system to take the oxygen to all the other parts of the body. So from your fingertips and your tippy toes, you know, to your brain, right? All over your body. We need oxygen, right? ,

Fatu Badiane-Markey  4:35  
Right right So the red blood cells are basically like delivery trucks, you know, right. They're delivering the oxygen everywhere that you need it.

Shekerah Primus  4:43  
Yes, exactly. Red blood cells are the delivery trucks. So it's like the mail van, right. Or maybe even the Amazon delivery van for those of us who like ordering from Amazon. Right? It brings that precious package that you ordered wherever you need it to be. So that's the job of the red blood cells in your body to get the oxygen wherever it needs to go in your body. So that's why when you start running or if you're exercising and you start to breathe faster, and your heart starts to pump faster, your pulse increases right so your blood is basically rushing through your veins, trying to deliver oxygen like more oxygen as quickly as possible to these muscles that you're using, that are using a lot of energy. Okay, so if red blood cells are the delivery truck that carries the oxygen, how is the oxygen packaged right, that's the next question. So when you order, say a new airfryer you need to get rom Amazon I can't help myself I gotta bring food in here, right? Or say you order, you know, some new yoga pants. It's got to come in some kind of packaging right to keep it safe in the delivery truck during transport. So in red blood cells, that packaging system is a protein called hemoglobin.

Fatu Badiane-Markey  6:16  
Yeah, I really like this analogy. I feel like it's so relatable, you know, because we all get packages. So tell us a little bit more about hemoglobin.

Shekerah Primus  6:26  
Yeah, so hemoglobin is the hero of our story, but unfortunately it's also the villain of our story. So it's the hero cuz yeah, it carries oxygen. You know, throughout our body. We need oxygen to live, but also it's the villain because when it's not working properly, it causes problems.

Fatu Badiane-Markey  6:50  
Okay, so can you explain that a little bit more?

Shekerah Primus  6:54  
Yes, ma'am. So sad but true. So I mean, we all know so as I said, hemoglobin acts as sort of the packaging, right for the oxygen that's carried in your red blood cells. So as we all know, packaging is not foolproof, right, sometimes, when you order a package, it gets dinged. You see that huge dent in the box, your heart drops, you know, you're disappointed you don't even want to open it cuz you just know something's broken in there. Right? Or if there's a hole in the package, you just know there's a huge stain in those yoga pants right girl, 

Fatu Badiane-Markey  7:30  
Oh girl, all the packaging nightmares 

Shekerah Primus  7:32  
All the packaging nightmares. So packaging is not foolproof. And unfortunately that is the case with hemoglobin in sickle cell disease. That packaging system is defective, and it's defective because it's mutated. Right? So the thing is, though, Fatu that, that in sickle cell disease, that mutant hemoglobin actually changes the shape of the red blood cells themselves. Right? So it causes the transport vehicle, the red blood cells to be deformed. That's intense, right? Imagine if you had a delivery van, carrying a bunch of packages that didn't fit well inside the van. Right? So maybe the boxes were too long and you had to drive but the back of the door open and the boxes sticking out the back of the door, maybe sticking out the passenger window right or maybe the packages are way too heavy. So it was weighing down the van so much that the you know your back exhaust pipe is scraping the roads or whatever, in both of these situations. I know it's Very, it's very like intense these situations are these situations. In both of these situations That delivery truck would have a hard time doing his job. Right. If you're driving with your back door open and packages sticking out. You have a pretty tough time going up hills and turning corners going over speed bumps, that kind of thing. So similarly, with sickle cell disease, the red blood cells are misshapen so they can't function properly. And instead they cause a lot of problems. 

Fatu Badiane-Markey  9:08  
Wow.

That's so intense. And it's you know, it's just it's really incredible to think also it's like, it's not even like the entire red blood cell that has the issue. It's just this hemoglobin protein. And that sort of leads this to this cascade of problems right? That then just make life so difficult and painful for patients who do live with sickle cell disease every day. 

Shekerah Primus  9:34  
Yeah.

Yeah. And that tells you how absolutely essential hemoglobin is. Right it has such a big effect on the red blood cells. I agree

Fatu Badiane-Markey  9:43  
Definitely, definitely. Yeah.

So you mentioned that red blood cells can't function normally when they're misshapen. So what symptoms does that usually cause in patients? Can you explain that a little bit more.

Shekerah Primus  9:58  
So normal red blood cells are disc shaped so think of, like CDs or DVDs? Those are kind of old now I know. Right? So what's new like blu ray discs right? I don't know what do they have now fatu some kind of disc or like a Frisbee right? Do people even playe Frisbee anymore? I dont't know. Think of some kind of disc right?

So sort of like a flat disc shaped type of cell. So in sickle cell disease, the mutant hemoglobin distorts the red blood cells into a sickle shape. So think of like a half moon or like a quarter moon right, some sort of crescent shape. Right. So this is also called a sickle shape. And these sickle shaped blood cells can't travel through the blood vessels as easily as the normal blood cells can because they're kind of stiff, right? They're not as flexible as say these discs that can bend and and you know, turn through the tight blood vessels as easily. So instead, these sickle blood cells tend to clump together and if you've got cells clumping together in your blood vessels, that's going to slow down blood flow. Right? And it can even cause blockages, right especially in those smaller blood vessels in like your extremities your fingers and toes, right. So these sickled blood cells also they break down easily. So there's constant loss of red blood cells, and that results in low a low number of red blood cells, which is called anemia. 

So people suffering from this disease are usually anemic and many conditions can cause anemia. Right? But anyone who's anemic will tell you that one symptom is feeling tired, so fatigue and shortness of breath. Also, with sickle cell disease, the rapid breakdown of red blood cells causes jaundice. And that's when you get yellowing of the skin and of the whites of your eye. They get yellow and that's called jaundice. So there are so many symptoms that people with sickle cell disease suffer from. And the symptoms vary in severity from person to person, but the lack of oxygen to the organs can lead to organ damage. They get painful swelling in the extremities, right their hands and their feet. They also can have high blood pressure, right? If you have something blocking your blood vessel, your heart is pumping as hard as it can to try to get blood to those farther parts of your body that is so hard to get to because there's a blockage, right. So they can have high blood pressure, which is another serious condition in itself that can lead to heart failure and strokes, right. And on top of that, they also tend to get a lot of infections. So this is an incredibly severe condition to live with. 

So these people can have normal days when they feel fine. And they can have days when they're feeling really tired, like we said, Right? Imagine going about your daily life climbing steps and doing all the normal things you usually do. But you're doing that all while not getting enough oxygen, right? So it's almost like your cells are gasping for oxygen so you're gonna feel exhausted. Right? And they can do so they can go from normal to tired to that situation I just explained where the sickled blood cells clump together, and they get stuck in the blood vessels and cause a blockage. And that the technical term or the medical term for that is vaso occlusive crisis or voc and that's a really dangerous situation as you can imagine, because it can cause eye problems, stroke and death of course, right it can of course lead to death. So and these people have excruciating pain, right from these blockages. So when this happens, they have to be hospitalized to get blood transfusions and treatment for the pain and to clear the blockage. So some patients with severe sickle cell disease describe having to be in the hospital every single month for treatment for these pain crises. And sometimes they're in the hospital for weeks at a time. So it's just an incredibly incredibly debilitating disease to live with.

Fatu Badiane-Markey  14:37  
Yeah, oh my goodness. I really I that's just really, really debilitating and so hard to live with. I couldn't even imagine that. And then I think the really scary part also is these things can happen anytime, right? So you can go from a day where you're feeling like you know, like, okay, you know, things are going well, then all of a sudden you have debilitating pain, and then you're in the hospital for weeks. I mean, how can you manage to live you know, a normal life and this is always going on in the back of your mind and then you have to then deal with all of these really intensive treatments. That's really really hard. Wow.

Shekerah Primus  15:17  
Yeah. Right. And actually, a lot of them reported not being able to live a normal life at all. Not being able to even have a regular job because if you're in the hospital all the time, right? I mean, yeah, so they it definitely disrupts their lives and causes a lot of hardship and fear and terror just I can't imagine living with that fear of oh my gosh, it could happen at any time, you know, so it's hard. It's really hard. 

Fatu Badiane-Markey  15:46  
Yeah, definitely.

So what percentage of the population suffers from sickle cell disease?

Shekerah Primus  15:51  
Great question. So sickle cell disease affects millions of people worldwide. And estimates range and the change as these things do but they range from about 20 to 25 million, with the highest incidence being in Sub Saharan Africa. And elsewhere, it's most common in people with African ancestry. And so living in the US we automatically think Oh, black people, right? But it's not just black people. It's also common in Mediterranean countries, such as Greece, Turkey and Italy, the Middle Eastern countries, Saudi Arabia and Lebanon, for example, India, and also Hispanics, regions in South America and Central America, and parts of the Caribbean. And it's also been seen in American Indians as well. So I mean, this is a genetic disease, right? So of course, it can affect anyone but these are the populations that it's most prevalent in. And in the US, sickle cell disease is the most common inherited blood disorder. And it's estimated to affect one in 500 African Americans and about one and 1000 to 1400 Hispanic Americans.

Fatu Badiane-Markey  15:58  
Wow. That's a pretty high prevalence if you think about it, especially in the US and so widespread, which I honestly didn't know. So, but yeah, just like what you're saying, since it's genetic, it's like once you have the gene essentially in that population, right then from there, it can go anywhere.

Shekerah Primus  17:32  
Yeah, exactly. 

Fatu Badiane-Markey  17:36  
So yeah, so that's a pretty heavy disease burden. Also, you know, with the numbers that you just mentioned, and patients can receive treatment to manage the symptoms, but there was no cure initially, right? So can you tell us how CRISPR gene therapy really changed all of that?

Shekerah Primus  17:57  
Yeah. So there actually was a possibility of a cure. Right? If and this is a huge, huge if, because it's if you could get a bone marrow transplant, right. I mean, just like with any transplant, finding a match, that's a huge problem, right? I don't even know what the numbers are one and whatever that you can find a bone marrow match to get a transplant and then that's a pretty intense procedure to you know, ask another person to do so usually it would come from like family members like a sibling or something like that, who was not affected by sickle cell disease. But the CRISPR gene therapy with the CRISPR gene therapy, you don't have to find a match, right? There's no match to look for because the therapy uses the patient's own cells. 

Fatu Badiane-Markey  18:54  
Oh, okay.

That makes sense. I got it. I got it. So how exactly does that work then? And how does the therapy work? 

Shekerah Primus  19:02  
Yeah. So this treatment was developed by CRISPR Therapeutics and Vertex Pharmaceuticals. And so back in 2019, is when things started to get really exciting for this sickle cell treatment, right. So from what I remember anyway, so that's early tonight, 2019 before COVID or 2019 BC as the kids say now.  Before Covid is now BC, I love it. 

Fatu Badiane-Markey  19:33  
Girl we''re aging ourselves Let's not worry about what the kids are doing. 

Shekerah Primus  19:45  
So, I think that, you know, that's when I first started hearing about it. And so So CRISPR therapeutics and vertex pharmaceuticals, they use this really clever strategy. So one thing that's really interesting about hemoglobin is that there are two forms of it. There is a form that we use in the fetus. And you know, when we're very young babies, and then that form gets turned off and another form gets turned on. And that is what we use throughout the rest of our lives. So the fetal hemoglobin form is not the form that is the mutant is not the form that is affected by the mutation that causes sickle cell disease. It's actually that adult form that gets turned on early in childhood. So it's so interesting, and I don't think we know yet why the body does this switch from fetal hemoglobin to adult hemoglobin. And so what these companies did instead of trying to fix the mistake, the mutation in the adult hemoglobin, instead what they did was they turned back on the fetal hemoglobin because it works right. Obviously, if you've got through development as a fetus, you've got working hemoglobin. So they just turned back on the fetal hemoglobin in these adult patients. Yes. It's very clever, right I thought it was a really clever strategy.

Fatu Badiane-Markey  21:16  
Oh my gosh, yes. My science brain is like literally exploded when you said like those four sentences. Keep going Shekerah. I'm on the edge of my seat now.

Shekerah Primus  21:29  
Yeh, yes. However, the entire procedure is super, super stressful on the body. So this is what they did. First, they remove the stem cells from the patient. So these are called Hematopoietic stem cells. Basically blood stem cells, right? They remove the blood stem cells from the patient. And they edit those stem cells in the lab to turn back on the fetal hemoglobin gene in those STEM cells. So in the lab, so outside of the patient, they do this editing, and because it's done outside of the patient, that's called ex vivo editing. So you have to think, well, this patient has a whole set of blood stem cells remaining that has this bad gene. Are they going to now put back in these new edited blood stem cells and have them in competition with the old mutated stem cells? What they actually do is, the person goes through chemotherapy to kill the disease causing stem cells in their body. And that is why this procedure is so tough on the body. Right? Because it's like having cancer, right? You have to go through chemotherapy. And so I remember reading about Victoria Gray in particular, she's one of the patients I've heard a lot about. She lost her hair, right? Of course, chemotherapy, right so she lost her hair. She went through like all of the typical chemotherapy symptoms and it's so hard on the body, you have to worry about getting infections, all that type of thing. So they said that the patient goes through chemotherapy to kill the disease causing stem cells in their body. And then the doctors put the edited stem cells back into the patient's body just using an IV. And the expectation is that these edited stem cell are they're going to find themselves to the bone marrow whether where they should be and they're going to colonize the bone marrow and start producing fetal hemoglobin. And I mean, I think that is so just so amazing. And it works. Right. So I think, a couple like a year after, when they were sharing their the results of the clinical trial they were saying that 30% of the patients hemoglobin was still this fetal hemoglobin, right and so they're still producing the mutant red blood cells. But now you've also got the fetal hemoglobin that's producing normal blood cells, so that these people can at least get you know they can get as they can. Get enough oxygen that they need throughout their body. And so what this does is this gives them the ability to live normal lives. They don't have these pain crises anymore, with you know, their blood cells blocking up their blood vessels. Because they have this normal fetal hemoglobin now to do the job of carrying oxygen through their body. So I just I just love that that they did it this way. And I think this is a really really clever way of doing it. And so patients with sickle cell disease, so I'm specifying with sickle cell disease, because this particular treatment is both for sickle cell disease and another disease called beta thalassemia, which is similar it's another blood disorder, but I'm specifically focusing on sickle cell disease, the clinical trial results that they reported is that the patients with sickle cell disease are free, completely free from transfusion. They don't need blood transfusions anymore, right? They have enough normal red blood cells now to carry blood through their body so they're completely free from transfusions and that disabling pain crises. The VOCs they used to have to be hospitalized for and stay in the hospital for weeks on end. So yeah

Fatu Badiane-Markey  25:31  
my gosh, in credible, like with the capital, I all and the capital cred and a capital in credible, I think this is one of the things that I just love about science, right? It's like taking a problem. We know it's there and Okay, we're gonna fix it. But a lot of times, of course with biology and like, you know what we see it's never straightforward. It's never like just A to B and you get C. But these researchers, you know, just were really ingenious and being like, hey, we have this fetal hemoglobin that we know works. Let's see if we can turn it on. And I think it's just amazing that it works just as well. In adults, as it does in the fetus. And yeah, and then you literally have a fix, right? And this is normal and it doesn't contain the mutation. It's just, it's just amazing. It's I have like no words. It's just amazing. I was literally on the edge of my seat the whole time that you were talking about all this because I was jaw on the floor, everything. It's just so incredible. And then you give these people their lives back, right. Yeah, so can you talk a little bit more about like the results and the feedback from the the clinical trials like have you mentioned you know, now they don't have to get transfusions? You know, they're basically free from the majority of the symptoms. It sounds like including, the really bad ones like the disabling pains, but so now can they also work and you know, just kind of like live normal lives. How are things going for Jimmy and Victoria? Yeah,

Shekerah Primus  27:15  
basically how to it's it's been a game changer. They're basically cured. Right from from this disease. It's not they're not cured, like genetically cured, right? Because they still have that mutation there affecting their adult hemoglobin, but they're cured because they're able to produce this fetal hemoglobin that can do the job of the adult hemoglobin. So it's like I don't need you no more adult hemoglobin

Fatu Badiane-Markey  27:44  
Take a seat

Shekerah Primus  27:46  
exactly. I will use my fetal hemoglobin. I think it's so interesting that our bodies does that. I really wonder, like, what was the evolutionary advantage to switching from fetal hemoglobin to adult hemoglobin, especially if the fetal hemoglobin works? Right? Right. So it's really interesting. I wonder what the advantage was, but yeah, basically, they're, they're cured. So in the very first episode of this series that we're doing the CRISPR Chronicles, we talked about Jimmy Olaghere, and Victoria Gray, who were part of the clinical trials. And so they have talked about how much does sickle cell disease CRISPR treatment has changed their life. And so I'll put a link to that episode in the show notes and then also tointerviews that Victoria and Jimmy have given so that you can hear directly from them describing the changes in their lives. But Victoria in particular talked about being in the hospital almost every month, and having to have an in home caregiver to help her to actually now being able to hold a regular job. And while a lot of us who have regular jobs might think this is awful, who wants a regular job.

These people this is like oh my god, I could have a normal life. I can have a normal life. It's like it's so amazing.

Fatu Badiane-Markey  29:14  
No, it's so true. I feel like sometimes we take for granted, right? of just what normalcy means. And you know, not everyone gets the luxury of having that, you know honestly. Yeah. All right. So amazing

Shekerah Primus  29:31  
that's an important perspective to keep in mind the next time you feel like quitting your job, you know, Not everyone even has the ability to do what you have the ability to do so. So the treatment has been a game changer and gave them the ability to live normal lives.

Fatu Badiane-Markey  29:51  
Just absolutely amazing. And so the treatment has already been approved by the FDA. So now is it you know, available for potentially other sickle cell patients?

Shekerah Primus  30:00 
Yeah, so it's approved both in the us and in Europe I think it was approved in the us December Eighth is the date I have in my head right now and then in Europe it was approved in February so a couple of months ago so yeah it's it's it's approved. They you know they can use it to treat patients with sickle cell disease especially those severe forms that have people in the hospital for weeks at a time. Um, and it's the first crispr-based gene therapy to be approved. So it's.Yeah, it's you know, breaking boundaries and and all that it's just it's so wonderful to have that and I think it sets a really exciting precedence and look forward I look forward for what's going to happen Next you know what's going to come next. What's going to get approved next how many diseases are we just going to start curing diseases left and right you know changing lives by the millions. That's that's such a wonderful positive hopeful thought I Love it.

Fatu Badiane-Markey  30:53  
Yes, no, it is so inspiring Shekerah and I'm just thinking like you know this is happening within our lifetimes you know I think that part of it is also just so incredible from when crispr was just like a theoretical thing to actually discovering it to having you know, um, researchers win the nobel prize for it to now having it used to actually treat diseases like that time is so short you know and it's so amazing and this is you know the first crispr-based gene therapy, and it's just going to lead the line to newer ones which I think are also going to make these treatments even more accessible to even more patients and it's just really the sky's the limit. You know I think it's just going to be so exciting to see what can come out of this and what we can do next and How we're really just going to be able to give people back normal, simple even maybe sometimes boring lives and that's okay, you know, like honestly because it beats being in a hospital for days on end and in excruciating pain you know and just being able to just have a 9 to 5 and just live you know a regular life and enjoy you know your family and friends and everything. So yeah, there's so many benefits to this and this is just it's so exciting like I can't wait to see what's what's coming down the line because if we did it for this I Mean who knows what's going to come next.

Shekerah Primus  32:24
Yeah, yeah, yeah, and you know what's so inspiring also just reading the stories about the people who live their lives with sickle cell disease right and to see how much they aspire to achieve a lot in their lives. And they have to deal with these pain crises you know and like being in the hospital all the time these really you know difficult situations but they're so hopeful and there's so much that they want to accomplish with their lives and now all of a sudden It's like oh my gosh The sky's the limit I can I can do all of these things. So um, yeah, it's it's It's very. It's very cool. It's been a really good. Yeah, really really good as you said to see this happen in our lifetime for sure.

Fatu Badiane-Markey  33:10
No completely, completely

Shekerah Primus  33:12
So that is it for this episode of the crispr chronicles. The next episode we'll have we'll do really light touches into some of the other clinical trials that are ongoing using Crispr gene therapy to try and cure genetic diseases and even some infectious diseases as well. So we'll have a really nice episode. It won't be as in-depth as this sickle cell episode was but it'll be you know, really light touches on some of the diseases that um maybe the most exciting ones that are Currently in clinical trials for Crispr Gene Therapy and so with with that we'd like to thank our wonderful listeners for once again tuning in to another episode of We Love Science if you'd like to reach out to us please do so by email at lovesciencepodcast@gmail.com and until next time bye everyone

Fatu Badiane-Markey  34:13
Bye everyone.