How Gene Therapy Works

stuff to blow your mind. That's the Julie Robert Julie doing their their science podcast, and we really we've talked about gene therapy a little bit on Tech Stuff in the past, we've never done a full episode on it. We've also talked about it on a sister podcast, the Forward Thinking Podcast all right, along with Joe McCormick. We we did a really great introduction of what jeans are and some of the really interesting cutting edge stuff going on in gene therapy. So if you would like to

listen to that, we we highly recommend it. We think that we did a pretty excellent job. Yeah. Yeah, the fact we had Joe there too. You know, put facts in between the jokes that I made and the shaking of Lauren's head. It really was different from a tech stuff episode. No, of course, I'm proud of all the work we do, and then one was a great show.

But we're going to give an overview of what genes are and some of the actual technical ways that scientists and doctors are working with gene therapy right now, because in the forward Thinking episode, it was more about the applications, right it was more about why you would use it and not how like how has it actually done? And

it's kind of fascinating. Yeah. Absolutely, and and this is, by the way, one of those things that we kind of struggle with all the time of of you know, we'll we'll come up with a concept for a show and then go, how should we check with car stuff? Should we check with mind? Should we you know? Who

should we ask? And make sure that we're not treading on their toes and re copying ourselves because we already did a forward thinking episode and really the answer is always, um, if it's interesting and there's more avenues within a topic to talk about, then then we'll go ahead and cover it. So let's start with the very basics. And but to start with the very basics, we've got to look at cellular biology. So anyone who was currently in middle school is going to be able to go on about this

at length. I actually had to go back and look up all this information because middle school was a long time ago for me. And while I was originally interested in going into biology, yeah I was, Biology was number one, and then it's always terrible most mostly that was not my branch of science anyway, Biology and physics I love them. And then I got into college and I switched to English literature with a focus on Shakespeare, and now I

talk about technology. So I mean it's a clear pathway. Yeah, logical progression so sells, you know, sells the basic little unit of life for for us, we human beings, and most of the stuff we tend to interact with that isn't super teeny tiny um cells have what is called a nucleus. It's kind of the brain center of a cell, the control center if you prefer, and they have a cell membrane, and both of these things are incredibly important

with gene therapy. So within the nucleus you've got chromosomes, and chromosomes are made up of two things, DNA and protein. And the DNA is the that's sort of the very basics of what makes you you. And along those chromosomes are little sections of DNA that we call genes now. The genes their job is to code for the production of particular proteins, and that's what we call a gene expressing itself. When a gene is expressed, it's coding for

a protein. And that's also important because with gene therapy, one of the goals is to introduce new genes into a cell so that a particular thing is express a particular protein is created, and it doesn't always work right.

So and the reason for that is because gene therapy this is still a very young science and uh and sometimes the techniques we use have high success rates, sometimes they have low success rates, and so it may be that you're doing everything right but or as right as we know how to do right now, but it's still not you know, success rate right since it's so experimental.

Right now, we're currently on using gene therapy to um to treat otherwise incurable diseases and particularly childhood diseases right and only in clinical trials for that matter, right. You you have to be part of a clinical trial, and the only gene therapy that is going on is being

conducted on somatic cells. Those are those are body cells as opposed to reproductive cells, right, which means that any changes that are going on, and and the kinds of changes that we're making are either inserting normal genes for herring abnormal ones or altering the kind of on off switches of those genes. Um they're only going to affect the patient, and any hypothetical offspring of that patient would not would not would not inherit those those genetic changes,

because again they're somatic, not the reproductive cells. So in another issue would gene therapy. Similar to that is that often the benefits that you experience with gene therapy are temporary in nature, and that you have to undergo multiple gene therapy sessions to have any lasting effect because they don't necessarily uh change the genome. It doesn't necessarily change everything like the actual uh way the cells are forming

from that point forward in your body. Right, It might just affect the current expression of that current cell, that current gene within that current cell, and if that cell dies, then you know that's the the benefit you got from that one cell is over. Yeah. I'll also, genes are really complicated. They most of them make more than one protein. That average is three, which is something that we've learned from the Human Genome Project, which I'll be talking about

a little bit later on. It just means that, you know, genes don't have they're not self contained bits of DNA, and they don't have strictly defined roles. Yeah, so you can't just say just get rid of that one, right, yeah, because it could be that a disease is actually the problem. You know, it might be the product of a few different genes, and then you have to figure out, well, which one of these genes is not behaving the right way, you know, which one is the mutated gene, which one

do we need to replace? And then by replacing that gene are we causing any other issues? Is there some other problem that could happen as a result of going in and altering a person's genes. And this is, like I said, a very young science. So we're still learning, and that's one of the reasons why clinical trials are the only place, at least in the United States where you can get gene therapy and you know, because honestly it's not ready for a full rollout yet, certainly not now.

So here we've got a cell, we've got that cell membrane. By the way, DNA does not pass through cell membranes. Cell membranes are semipermeable, right, which means that they allow some stuff through and they allow they keep other stuff either inside or outside. Now, DNA generally speaking, is something that cells want to keep inside. They don't like letting

the DNA go flying out all over the place. It's a better idea, yes, So that means that if we want to introduce DNA into a cell, we have to kind of find a way of getting the DNA through the membrane without damaging the cell because clearly or the DNA, So if you kill the cell in the process, that that's not what we call youthful. We call that. We call that a fail that's that's generally speaking, that's a failure.

You don't you don't want the cell to die. So finding a way to get DNA material into a cell, So getting a good gene through that cell wall without killing the cell has been really the main focus of genetic therapy over the last couple of decades. So there are a lot of different ways of doing it, and we could either go about this chronologically or we could

try thematically or maybe alphabetically. But we kind of decided like the first one we wanted to look at is in a way, sort of the simplest, at least in concept, if not in practice. And I guess the we're we're going to cover two basic categories UM in this podcast today and a couple of sort of outliers and and right, there's a bunch more. We'll talk about that a little bit at the tail end of the episode, and I

guess that we're sort of going chronologically a little bit. Yeah, it's a it's a little bit of a jump around. But but the first one we wanted to talk about is micro injection, which is more or less what it sounds like. Yeah, you're talking about a very very tiny needle going into a cell and injecting DNA material directly into either the cytoplasm, which is the the stuff inside

a cell membrane just in general. That's that's the uh, both the fluid and all the little organelles and everything that are in a cell, or you're injecting it directly into the nucleus, which is where the d n A material is kept. So in other words, you are using a needle and you're just inserting the DNA directly there. Now, when you're talking about cell cells are I don't know

if you know this, they're tiny. They're they're really little. Yeah, you might think that, uh, the needle that you're getting for techna shot is huge. Well it is pretty big, but to a cell, that's gargantuan and I'm nearly positive that many cells could fit inside. Yeah, so you need to find a very very fine needle and you have to be able to make. Really, you're not You're not

gonna You're not gonna just stumble over one of those. Okay, well, I mean, you know, if you happen to stumble in a lab, you might, but I accept that you make an incredibly fine needle, and you actually you kind of need to. One of them is just less fine than the other one. The element is more of a pipette, and the reason for that is you have to immobilize the cell that you want to inject. You need to kind of hold it down because otherwise cells are sort

of squid squiggly and yeah, they are. They do tend to squiggle. Okay, So so this this technique was really pioneered. The needle micro injection in general was pioneered by one doctor Marshall Barber in the early nineteen hundreds. UM. He was developing it to study bacteria and confirm the germ theory that was being developed around that time by a Cotchin pastor UM who would who would basically, um outline the entire germs make a sick kind of contract, which

was revolutionary at the time. Right, it's still I'm still pretty glad that we have it, and I think I think it's great. So early on, like through the nineteen sixties, early on, I mean, this was a very slowly developing field of study. Uh. Microinjection was used to implant whole organisms and embryos into larger bodies. Then in the nineteen seventies we started implanting cellular organelles and molecules UM and

other relatively large bits of stuff into cells. And then as we got into genetic study in the mid eighties, we began injecting stuff like proteins and DNA and RNA into cell nuclei. Right, So it obviously took quite a while. And the general process, again at least conceptually, is simple. You hold the cell still and then you inject the stuff into it. Yep um, it's it's all. It's all the same process, just just as a miniaturization of these

technologies began to get gooder. Yeah, right, because because otherwise holding a cell still that's actually pretty tricky. They use these pipettes that use a little bit of suction that immobilize the cell, and then you have to have a micro manipulator that's something that allows you to make very very precise movements because you obviously you could not use a handheld hypodermic needle. You wouldn't have the precision to be able to to target specifically the nucleus with any

level of confidence. I was, I was pretty good at duck Hunt, and I've got to say that I would not that I don't have that kind of precision. The hand eye coordination is pretty pretty uh remarkable. So yeah, they usually you have some sort of device that is designed to to study the needle and direct it properly.

So has limitations. There are some big ones actually. Also in order to really use this technology, we we would have to start getting into them what we learned through the Human Genome Project, which um, which didn't really get started until the nineteen nineties. I mean, it came about after the Atomic Bomb Project in World War Two, actually because Congress charged the Department of Energies predecessor agencies with UM studying and analyzing genome structure, the replication, damage and repair,

air and and consequences of genetic mutations. UM. So you know, especially those that were caused by radiation and the chemical byproducts of warfare energy production. Right. So, UM, the Genome Project completed its initial research a couple years ahead of schedule in two thousand three. And UM, that's basically where where we got most of our information. You know. That that that that is the spawn, that the dawn of

gene knowledge, right, you know. Without that, obviously, we couldn't make any um, any knowledgeable decision about how to go about this at all. Right, we wouldn't know what was wrong, much less how to correct it, all right. And so that's that's why really everything that we're that we're talking about has happened in the past like two decades. Yeah, it's pretty exciting stuff. So this one does have micro injection,

does have some drawbacks. A big one is that you can't really conduct this UH in vivo, meaning within the body of a patient, because you can't you can't immobilize the cells. You can't really use the micro manipulator to inject something directly. All this kind of stuff tends to be done in a laboratory, in a special lab dish in the glass, right, Yep. You usually have a solution and you end up using a little pipette to immobilize the particular cell you want to UH to to manipulate.

And now a lot of times they would use bacteria for this sort of thing, the idea being that the bacteria would then transmit the DNA to other cells. But we'll talk more about that and probably a future podcast. It's it's really kind of immaterial to this UH. There was another downside. It's that you have to do it to one cell at a time, which is not terribly efficient. Nope. And another UH drawback to this approach is that you have to be really careful exactly where you inject that DNA.

They found out that if they injected the DNA into the cytoplasm, it was far less effective than if they injected it directly into the nucleus are possibly because the electro light salute in the cytoplasm was degrading the DNA before it could do anything useful. Right, So, you know, just like we said earlier that just because you put a new gene into a cell doesn't necessarily mean you're going to see that gene express itself or be effective. But they found that there was much more effective if

they did inject it directly into the nucleus. Uh So, I mean it's it's certainly useful within the lab, but within real world quote unquote settings where you're trying to work on a patient directly, it's not. Now, if you can develop an approach a cure like within the lab within there and then implant those cells into the patient,

that might be a way to go. But in general, this tends to be something we think about in terms of learning more about what's working and what's not working, as opposed to an approach um actual therapeutic treatment exactly. So that kind of leads me to another one hydro dynamic pressure, which again sounds you know, it's pretty much what sounds like, I didn't read that much about this one.

Tell me, tell me what it is, all right. So they found out that if you take a relatively large amount of DNA material, and so for a human we're talking about actually just a few millilets, but that's relatively huge amounts of DNA, and then you take a needle and you insert the needle into a blood vessel inside that person, and then you inject that relatively large amount of DNA very quickly into that blood vessel. Then this the hydrodynamic pressure will cause cells along the blood vessels walls,

the indothelium, that's the layer that the lining. Uh, it'll cause them to become more permeable, as well as the perenchyma cells or paranchema cells. Now these are like tissue cells. So this is like the cells of stuff that actually does work depending upon whatever you know, Oregan, you are targeting. So let's say it's the liver, all right. Well, they

found that also increases their permeability. So if you have that DNA material and you inject it very quickly into the blood vessel, then you can end up having a lot more of that. Yeah, it ends up a lot more cells are going to accept that DNA and potentially you will start getting that gene expression, you're looking for. UM. Couple of other downsides to this. Now they've they've been

using this with lab animals, not with people. All right, so this is not something that's been tested on people, but they've tested on mice and rats and it never got to the human stage because well, because they found out that sometimes this causes some cardial problems in the car cardial and respiratory. Yeah. Yeah, So when they would inject the vein along the tail of these critters, the

mice and the rats. They did it with a mouse, the mouse would start to seem to have trouble breathing for a couple of minutes and then recover and everything seems to be okay. Rats could sometimes stop breathe entirely. And they found that by massaging the abdomen's they could, uh, they could they could end up getting the rats to

you know, kind of start breathing again. But they discovered that using this approach put a lot of pressure on the animals uh cardiovascular system, and that it would cause everything from labored breathing to uh, irregular heartbeat. Uh. They also discovered that the animals livers were expanding, sometimes up to two of their original size. I guess that weirdly makes sense. I mean, in terms of of liquid essentially,

you're essentially overloading the system, right, you know. And uh, and so none of these um issues were permanent unless, of course, the animal did not survive that initial reaction. But the animals survived, then their various systems would return to normal, Like they're breathing and heartbeat would return to

noble normal within a couple of minutes. Uh. Their liver would go back to being normal sized within about half an hour, and some of their other systems would take up to two days or a day and a half or so to get back to normal, but they started to. They also would very frequently start expressing whatever the gene was, so in front that side, it was a success. So all of these so far have been interesting, UM, but perhaps not the best way to actually treat human patients. UM.

We've got a couple more to talk about. But first, Yeah, let's take a quick break to thank our sponsor. All right, we're back. So we've talked a little bit about a couple of different approaches that may we may never see used as far as human applications go. I mean, there are there's work in hydro dynamic to try and make it a something that we could use with human patients.

But anything that would put that much stress on your cardiovascular system for someone who's already trying to undergo medical treatment is definitely not going to be the first choice, right right, No, So let's talk about um, well, let's talk about zapping cells. I was just thinking, you know, if we can't if we can't really stab a needle into a cell, if it's inside a person, and if we can't really expect to jab a bunch of liquid into a blood vessel and possibly cause a heart attack,

maybe we could just shock the heck out of them. Yeah, that's clearly the next logical step. Um no, no, but actually it does make a lot of sense. Yeah, because I mean, we've got those membranes. They're not going to let that DNA just go right on in. So how do we convince it that we need to get this DNA from outside the membrane inside the membrane? Right? Okay? And one of one of the really cool and important facts about cells that that you that you need to

realize here is it. Cell membranes are basically insulators that are separating two charged regions, the the electrolyte or ionized solution inside the cell and the electrolyte solution outside the cell. So so they can they can act as capacitors. They have they have capacitance. They do have capacitance, and if

you overload that capacitance, things go wonky. Stuff happens, right, So, if you do it at the right amount of of charge, then it ends up creating these pores, these these temporary pores p o r e s. So you get these little holes that form in the membrane, and it's temporary. They will actually heal back up actually actually just a few minutes. Yeah, within a few minutes after zapping them, assuming that you're zapping them with the right amount of electricity,

because that's really important. So if you if you do it at a low enough UH or well really it's short high voltage zapps um very low amperage, and so you're just doing these tiny little controlled zapps to the the cell membrane, these holes will open up and then within a few minutes after you've applied it, they'll close

back up again. So while they're open, then you can coerce d NA to go in because DNA's are charged particle and right so so especially since you've just changed the capacitance of that membrane, it makes it easier for stuff to slip through when it's also charged. So you just you kind of you kind of corral it in so you're you're you're driving the DNA and it's not

like it's just gonna you know, oh yeah, zip. You actually have to guide it yourself, yourself being if you're a scientist or doctor who's doing this sort of thing, obviously probably not like no, no, no, no, probably would not do this. I think Noel does want to stab me with stuff, but probably not in a medicinal way. Anyway. This is called electroporation. Obviously you're you've got the electro

and the pooration. You've got the making the poor. So it all makes sense when you know what's going on. This was originally investigated in the nineteen sixties, so doctors began to experiment on cells within the lab that you know, they're not looking at in vivo yet working in the lab, and they were overloading the capacitance of the cellular membranes. They were just introducing small amounts of electricity. Actually, originally, what they were doing was They were using equipment called

electrophoresis apparatus. An electrophoresis is the manipulation of charged particles through a solution. So if I have a solution that's neutrally charged, and I put some charged particles in it, like some negatively charged particles, let's say, and then I generate a negative charge and move it close to those particles, it's going to push them away, right, because like charges repel one another. So you can actually move stuff through a solution this way. And often in chemistry this is

used to separate materials out from a solution. And in this case, it would be too convince d NA that needs to go through that gigantic gaping hole in the cell membrane right in front of it. And uh so what they did was they took this equipment that was meant to just guide charged particles, they short circuited it on purpose and tried to use it to zappa cell wall. So obviously, with this approach, it wasn't what you would call precise. So but they they saw what the effect was,

but it wasn't fully controlled. Once once they got it more under control, though, oh yeah, yeah, because they did discover that it could affect the cell membrane. But they also discovered that you could fry a cell. But if you were able to get just the right amount of electricity at the right frequency of pulses, you could cause the cell membrane to create these pores and not harm anything inside the cell itself, which of course is obviously important.

You don't want to kill the cell that you're trying to introduce the new gene into. Right Again, that is what we call a fail. Yeah, not not something you want to have happen. So uh for a long time they would work on this uh in vitro. So again in the glass, they would have a glass of cells. They would use a circuit that would encircle the solution that the cells were inside. And this solution in this case would be a conductive solution, meaning that it could

also conduct electricity. You turn on the electricity for the right amount of time causes these pores to form, and then you would corral the DNA into the pores, and then you would check to see if the genes that you wanted to have expressed in those cells were in fact expressing themselves, either either making proteins or or etcetera. If they weren't, you made them listen to Madonna over and over until they got the message. I had to make that joke somewhere, Lauren, it might as well be here.

You've got to make it fair enough, all right, So we allow you the Madonna pun. Thank you. So this was really promising, I mean, and in fact, it's promising beyond gene therapy. That's just one application of this particular technique. Right, These next couple of things that we're talking about UM are also really effective for UM for introducing drugs into Yeah, so let's say that you have a tumor and you need to have some sort of chemotherapy delivered to it. Now,

tru sational chemotherapy is essentially affecting your entire body. You you are poisoning yourself. You're doing in a very controlled way. But yeah, and it's and and it and it can be absolutely debilitating. Yeah, I've got friends who have gone through it, and and it is it's hard to see. So if you're able to to introduce that medicine directly to the cells that you want to target, you can

target a very localized area. UM. And and since the nineties, I think they have been researching how to do this in in the body and yes, yeah, in vivo and uh. And so that could mean that you have a very much more effective treatment that has fewer side effects. It doesn't eliminate the side effects, but it might make them less traumatic for the patient. So the en vivo approach is you might wonder, well, how could they use this

electrical uh technique within the body. And it's not entirely pleasant, folks. They usually use either tiny electric plates or electrified hypodermic needles. Oh man, you're saying all the words that I want happening to my body better than cancer. Yes, So these they would they would go to the location of wherever it was they needed to introduce the genes. So let's say it's your liver again, because that's it's a large oregon.

It's one that it's the one they think that they'll have the most success on early because it's a large oregon that's relatively easy to manipulate compared to some of the others. So yeah, they would. They they would have to either surgically insert these plates or they would have to use these needles to introduce the electric current. Yeah, and to to create the field exactly the way they

want to to introduce the DNA material into your system. Um, it's pretty invasive, and that's one of the reasons why there are some researchers who are trying to find some other method to introduce gene therapy that's that wouldn't be as invasive as this approach. All right, I think I think didn't have a pretty bad reputation when it was when it was still in the in vitros. Yeah, you know,

it wasn't. It wasn't great. Um, they were working with bacteria, and they were killing bacteria cells pretty frequently, and they really just needed one cell out of the batch to live. So right, if you're like, if you're like, there's a I gotta I got a couple of thousand cells in this in this dish and only one of them has to live, you don't necessarily go easy with the big old switch, that says fry Um. And so it did get a reputation for being a violent method of gene

therapy delivery. But then that's because they were working with bacterial cells, not necessarily working with like and it wasn't it wasn't in a patient. Yeah, if they could had to be a little bit violent, right, but give it a bad reputation. Yeah. So then you've got patients who are like, I really don't want something where you're, you know, essentially putting a cell sized electric chair into my body.

Please can we do something else? So yeah, there there's another approach that essentially creates the same effect, but it does it in a totally different mechanism. Right, sound operation, yes, son operation so saw No, you start to think that sounds like it might have something to do with sound, and it does. It actually uses ultrasound. So you use ultrasound at a particular frequency directed that the cells, and the cells will end up forming these pores just as

they would with electroporation. Right, This was really pioneered in in this first decade of the century. And uh, basically, you put stuff that you want to get into a cell into what's called a microbubble or a bunch of microbubbles really, so exposing cell to to this ultrasound will create the pores, changing the membranes conductivity and making it

easier for stuff to slip in. Right, and um, the same ultrasound will also burst the microbubbles, which will release the st up that you want to get into the cell in a place that makes it easy to kind of push in there. So interesting, So you have not only not only is it opening up the doors, but it unleashes the stuff that you want to deliver to the cell itself, and you don't have to worry about it being absorbed by the body some other way before

it can get to where it needs to go, right right. Um. It can also burst the entire cell if you're not careful, so that's the thing that you have to watch out for. Bummer. Yeah, although from what I understand a lot of scientists refer to this as being much less invasive than the electroporation in vivo approach. Oh sure, sure, well, and any time that you're talking about ultrasound, it's something that can be

applied um externally, Yeah, which is pretty cool. I mean I've actually seen ultrasound also being suggested as a means of directing nanoparticles to go straight to particular cells. So it's like having a nano sized r C car moving around the body. Doesn't work unless you're Martin Short and you've got uh Quaid inside of you. Dennis Quaid is inside of you, you know what I'm talking about. I I know, I know inner Space, Yes, I'm familiar with it. I'm I'm just thinking about the that that old Epcot

Red Body Wars. Yeah, that was in the old Wonders of Life pavilion. That is sadly no more. I'm not sure. I'm not sure if I'm sad about that. That was not my favorite pavilion when I was a kids. Was great. Cranium Command even better. Guys, all right, Disney fans out there who know what I'm talking about with Cranium Command, right in and tell me whether or not you loved Cranium Command, because honestly, that is one of those attractions

I wish Disney would bring back. Also, if anyone here happens to say work in the advertising department of Disney, um call us because we we will clearly do Disney as Yeah. So getting back into the actual gene therapy. So there are other methods we could discuss. There are a lot of things called viral vectors. And you might wonder, why why would you ever use anything that has the word viral in it. Well, well, there's there's a bunch of different of different vectors that you can use. Um

and and and viruses are one of them. And this is so fascinating to me because you're using really virulent viruses and the kind of like like HIV or herpies from in order to you kind of scrape out all of the harmful DNA contained inside of a virus and put in whatever you want to get into a cell, right, and that sometimes can be RNA, can be DNA, or it can even be a chemotherapy drug like we said before. And the proteins on the outside of the virus, on on the virus shell itself act as kind of like

a homing system, right. It will only end up interacting with particular types of cells, because that's what viruses are. They are they have this this uh, this the structure that has them interact with very specific types of cells. And they're very good at doing that. They're they're really good at at invading a system, finding the kind of cells that they that they want, and um and and

and stuff in there replicating like crazy. Yeah, So in this case, instead of replicating, you would put in the material that a virus need to deliver, either genes or whatever you wanted. We'll talk more about that in the future episode, because that's an entirely huge topic that we

could cover. Yeah. That and there's also um what's called these biodegradable nanoparticles, which is a similar a similar vector system in which you're directing um charged particles that are going to go to a specific place and do a specific thing. But um but instead of being kind of piggybacking on a virus, you're you're doing it with with nanoparticles that can be can be controlled through the properties

of those particles gotch. So it's so it's a synthetic approach that kind of mimics what the virus does, but allows us to have an external control system where we say this particle we want to go here, and then it can do what it needs to do. There are other approaches that we could talk about and probably will talk about in future episodes. For example, impale affection. Uh does this have anything to do with staking vampires? It does not. Uh, Buffy had nothing to do with this, No,

But it's pretty much what it sounds like. You know, you're impaling stuff, and you've got the right material on whatever it is you're impaling. So let's say that let's say that you're you've got to sell that's the size of I don't know, Spike and you've got you've got a delivery system that's the size of Buffy and the steak is covered in in uh in in strawberry jam, and she steaks Spike and now he's got strawberry jam in his system. That's essentially what this is. But we'll

go into more detail in the future episode, I promise. Um. And there's also a laser beam gene transduction. We could go into that, but that's another one of those that's sort of on the developing side, and I thought that that would really lend itself well to a second episode

if we decided to do that further down the line. Um, we really going to concentrate on the these are these physical exactly the ones where people are using technical tools right now to experiment and see which delivery systems are the most effective. Because again, just like just like I said with the very first one and every single one of these approaches, even being successful in introducing the DNA to the cell, does not guarantee that you're going to

get the result you want. So it's one of those things that we're still learning those basics. Yeah, and you know,

it's it's really it's really scary. Um, and a lot of the things that I've that we've talked about, I feel like we have ended on notes of of like and that's terrifying because, um, but it's also incredibly promising, you know, like I said at the beginning of the show, especially for children that have incurable, terrible genetic diseases that really have no other way of receiving therapy, this is this, This is their best shot, and it's wonderful, right, Yes,

And while we're talking about just like you were saying, like these high risk approaches, the risk is going down over time because we learn more and we learn how to how to apply it more effectively. So I personally think this is a very promising area. And you know, we didn't even touch on gene therapy to do stuff like give you crazy awesome pecks or something, you know, like yeah, none of the sci fi. Yeah, there's there's plenty.

We could do an entire episode about that too. About all right, well, let's assume that we have perfected gene therapy applications. Now we're able to go beyond just the medical approach and we all either um, look like ethan hawk or can shoot bees out of our hands. Yeah, dogs with bark and bees come out. Um, fantastic. Well, I look forward to our wicker Man future. Uh no, this this really is an interesting topic. It was certainly something that I I found fascinating as I researched more

and more about it. Because I had heard the terms. I knew in general what was going on, but I didn't know from a technical goal level or even a cellular level, exactly what was happening. And it's pretty exciting stuff. So I'm very interested to see how this field develops over time. I'm hoping within our lifetime we start to

see some of these diseases just get eradicated through gene therapy. Alright, so guys, if you have any suggestions for future episodes, you've got some sort of topic you think we should cover, and maybe your name is Mike and you know how to use Twitter, you can get in touch with us on Twitter, Facebook or even Tumbler. We are tech Stuff hs W at all three of those locations. Or you want to go old school and email us, Hey, you

can do that too. Our email address is tech stuff at Discovery dot com and Lauren and I will talk to you again really soon for more on this and thousands of other topics because it has staff works dot com