In last week's blog post, I discussed the use of gene therapy as a treatment, and possible cure, for Parkinson's disease. At the end of the post, I mention my wish of seeing Muhammad Ali, who I refer to in the post title as the 'greatest boxer of all time,' cured of Parkinson's tremors. Well, I couldn't help but follow up on that post when I saw a brief article in Popular Science about an 8-year-old boy whose eyesight is restored in an eye treated with gene therapy. According to the original article in The Lancet, patients of the study were treated with a vector that encoded a protein that controls the activity of the retinal pigment epithelium, restoring eyesight in six of the twelve patients in the study with congenital blindness. The results indicate that, by studying both children and adults, the greatest improvement in eyesight is achieved when patients are treated with gene therapy at a young age, as the children of the study fared better than the adults.
Despite being somewhat void of details, the Popular Science article did have something that the original Lancet article did not: a video of the 8-year-old boy navigating a maze first using only his control eye, which did not undergo gene therapy injections, and then using his treated eye, which did. The difference is quite obvious. In fact, the first video of the boy trying to navigate the maze using his control eye was somewhat difficult to watch, as he had to be assisted at every turn. He even struggled to find the door knob on the door at the end of the maze. On the other hand, the video of his attempt using the treated eye is much easier to watch. Let's just say, if it had been a test, he would've aced it.
Very cool study and very promising results.
"But what's with the weird title" you ask? As I stated earlier, last week's blog post title makes reference to Muhammad Ali as the 'greatest boxer of all time,' and since I'm following up on that post, I thought I'd continue the theme. For those of you not in the know, the 'greatest living guitarist' refers to Jose Feliciano, who was born permanently blind. At 64 years of age, it's unlikely that Feliciano's vision will ever be restored with gene therapy, but it's appealing to think of a future in which congenital blindness has become a thing of the past.
Monday, October 26, 2009
Monday, October 19, 2009
Gene Therapy and the Greatest Boxer of All Time
Parkinson's is a nasty, nasty disease. There is no doubt about it. Of course, any disease that is described as both 'chronic' and 'progressive' is not going fun to deal with. A degenerative disorder of the central nervous system, Parkinson's disease (PD) is currently incurable, and effective treatments are nearly non-existent.
And this is where gene therapy comes in. Gene therapy has been considered the holy grail of Parkinson's disease treatment for quite some time. Studies that have focused on use of gene replacement therapies in neural cells have shown promise, but have lacked major efficacy, which would be required for large-scale, multi-phase clinical trials. Various gene therapy strategies have been pursued, all showing positive results, but would still leave PD sufferers with continued standard therapies that can often be cumbersome, expensive, and sometimes nearly as debilitating as the disease itself. A new gene therapy, which takes three previous gene replacement strategies and combines them into one, is being reported by Nature as a potential breakthroug in PD treatment.
It makes total sense...take three different types of gene therapy treatment that have shown moderate success and combine them into one single vector. A multi-functional attack that is aimed at rehabilitating the neural cells in three ways at the exact same time. Genius. And it gets better. The therapy has already been tested on humans, and is expected to move into phase II clinical trials soon. I would love to see this treatment become standard, not only because the eradication of Parkinson's disease would be a beautiful thing, but also because it would solidify gene therapy's place as a viable treatment option for other diseases. I mean, let's face it, there's still a stigma that the public has with gene therapy. And not all of it is without reason. Gene therapy has its risks, one of the largest of which is the insertion of the target gene into a place in the genome that causes major damage to cell function or, even more likely, that causes cancer. Another issue is the current trends in gene therapy research. Everyone would like to see the creation of a treatment for PD, but what about gene therapy treatments for colorblindness or baldness? By comparison, research into these kinds of gene therapy treatments seems a little less awe-inspiring. But hey, who am I to judge? After all, gene therapy research on colorblindness could open the door for other types of ocular disorders, and curing baldness is not always just a matter of vanity; for some, it really is a quality of life issue.
Anyways, I digress. What it really comes down to is this: I want to see the great Muhammad Ali standing tall and proud, without the devastating tremors of Parkinson's racking his body, speaking about what it was like to reclaim his title as World Heavyweight Champion from George Foreman in Zaire during the 'Rumble in the Jungle.' And maybe, just maybe, gene therapy could be the key to granting me this wish.
And this is where gene therapy comes in. Gene therapy has been considered the holy grail of Parkinson's disease treatment for quite some time. Studies that have focused on use of gene replacement therapies in neural cells have shown promise, but have lacked major efficacy, which would be required for large-scale, multi-phase clinical trials. Various gene therapy strategies have been pursued, all showing positive results, but would still leave PD sufferers with continued standard therapies that can often be cumbersome, expensive, and sometimes nearly as debilitating as the disease itself. A new gene therapy, which takes three previous gene replacement strategies and combines them into one, is being reported by Nature as a potential breakthroug in PD treatment.
It makes total sense...take three different types of gene therapy treatment that have shown moderate success and combine them into one single vector. A multi-functional attack that is aimed at rehabilitating the neural cells in three ways at the exact same time. Genius. And it gets better. The therapy has already been tested on humans, and is expected to move into phase II clinical trials soon. I would love to see this treatment become standard, not only because the eradication of Parkinson's disease would be a beautiful thing, but also because it would solidify gene therapy's place as a viable treatment option for other diseases. I mean, let's face it, there's still a stigma that the public has with gene therapy. And not all of it is without reason. Gene therapy has its risks, one of the largest of which is the insertion of the target gene into a place in the genome that causes major damage to cell function or, even more likely, that causes cancer. Another issue is the current trends in gene therapy research. Everyone would like to see the creation of a treatment for PD, but what about gene therapy treatments for colorblindness or baldness? By comparison, research into these kinds of gene therapy treatments seems a little less awe-inspiring. But hey, who am I to judge? After all, gene therapy research on colorblindness could open the door for other types of ocular disorders, and curing baldness is not always just a matter of vanity; for some, it really is a quality of life issue.
Anyways, I digress. What it really comes down to is this: I want to see the great Muhammad Ali standing tall and proud, without the devastating tremors of Parkinson's racking his body, speaking about what it was like to reclaim his title as World Heavyweight Champion from George Foreman in Zaire during the 'Rumble in the Jungle.' And maybe, just maybe, gene therapy could be the key to granting me this wish.
Monday, October 5, 2009
The Sky is Burning!
A team of scientists at the High-Frequency Active Auroral Research Program (HAARP) facility in Alaska has published the results of a recent study in which they induced the formation of ionosphere-like plasma within the lower atmosphere of Earth. I'll explain why this is so freakin' cool momentarily, but first I thought I'd briefly share why HAARP itself is worth discussing.
HAARP's purpose, as stated in the Enironmental Impact Statement is as follows:
And this is where things get interesting. In the recent study, which was featured by Nature, researchers were able to crank up the IRI and direct it at the upper atmosphere. The high energy from the IRI's RF signal excited molecules in the atmosphere to the point that they ionized, creating an artificial patch of ionosphere. So what does this mean and why does the government care about creating artificial ionospheres? To answer this question, I will conclude with a scenario that illustrates a potential application of this ability. Let's say that you work at a CIA base of operations in Eastern Europe. You have gathered top secret data that must be relayed to another base in the Middle East. Because there is not a direct line of sight between the two bases, the communication signal is sent skyward and bouced off of the ionosphere to reach the other base in a process called over-the-horizon communication (the CIA has been using this phenomenon for communications and intelligence gathering for decades). However, the angle at which the signal must be sent is fixed (because the ionosphere doesn't move) and enemy intelligence is monitoring transmissions occuring in this region. So instead, you use an RF transmitter to induce an artificial patch of ionosphere, which you bounce your communication signal off of. The angle of transmission is different than expected and the enemy is unable to intercept the signal. Your information remains secret. You're the big hero.
The end.
P.S. Like the Large Hadron Collider, the construction and power-on of HAARP created a fair amount of panic and doomsday scenario talk. It's worth noting that instead of killing us all, the facility has produced a constant feed of information and a wealth of knowledge on the upper atmosphere. Of course, HAARP has not destroyed the Earth. Neither has the LHC. At least not yet...
HAARP's purpose, as stated in the Enironmental Impact Statement is as follows:
The High-frequency Active Auroral Research Program (HAARP) is a congressionally initiated program jointly managed by the U.S. Air Force and U.S. Navy. The program's goal is to provide a state-of-the-art U.S. owned ionospheric research facility readily accessible to U.S. scientists from universities, the private sector and government...The program's purpose is to provide a research facility to conduct pioneering experiments in ionospheric phenomena. The data obtained from the proposed research would be used to analyze basic ionospheric properties and to assess the potential for developing ionospheric enhancement technology for communications and surveillance purposes.The facility consists of what's called the Ionosphere Research Instrument (IRI), which is an array of extremely high-power, high-frequency radio frequency (RF) transmitters. The IRI fires electromagnetic radiation into the ionosphere (the same thing that the Sun does, just from the opposite direction and in a highly controlled manner) and then uses sophisticated equipment to monitor the effects. The main idea of all this is to better understand how RF and other signals, such as those that allow us to communicate with our various sattelites, interact with the ionosphere so that we can use these properties to our advantage. This equipment is also capable of altering the properties of the ionosphere for short periods of time. For instance, by exciting the ionosphere with the IRI, researchers are able to imitate an aurora, a phenomenon in which charged particles from the ionosphere impact molecules in the atmosphere causing them to emit light.
And this is where things get interesting. In the recent study, which was featured by Nature, researchers were able to crank up the IRI and direct it at the upper atmosphere. The high energy from the IRI's RF signal excited molecules in the atmosphere to the point that they ionized, creating an artificial patch of ionosphere. So what does this mean and why does the government care about creating artificial ionospheres? To answer this question, I will conclude with a scenario that illustrates a potential application of this ability. Let's say that you work at a CIA base of operations in Eastern Europe. You have gathered top secret data that must be relayed to another base in the Middle East. Because there is not a direct line of sight between the two bases, the communication signal is sent skyward and bouced off of the ionosphere to reach the other base in a process called over-the-horizon communication (the CIA has been using this phenomenon for communications and intelligence gathering for decades). However, the angle at which the signal must be sent is fixed (because the ionosphere doesn't move) and enemy intelligence is monitoring transmissions occuring in this region. So instead, you use an RF transmitter to induce an artificial patch of ionosphere, which you bounce your communication signal off of. The angle of transmission is different than expected and the enemy is unable to intercept the signal. Your information remains secret. You're the big hero.
The end.
P.S. Like the Large Hadron Collider, the construction and power-on of HAARP created a fair amount of panic and doomsday scenario talk. It's worth noting that instead of killing us all, the facility has produced a constant feed of information and a wealth of knowledge on the upper atmosphere. Of course, HAARP has not destroyed the Earth. Neither has the LHC. At least not yet...
Monday, September 28, 2009
How To: Destroy a Planet
With the wrapping up of, from what I've read, a somewhat useless G20 summit, I thought I would take a quick stab at the highly complicated and politicized subject of global climate change. I was reminiscing about an article that I first heard on my local National Public Radio station. The story was about a 16-year-old girl who had become the poster child for skeptics of global warming. If you're interested, you can find the story here, or if you really want a laugh, check out this totally ridiculous version. The young girl had written several papers that tackled issues of climate change, with the main theme of her work being that current models of climate trends are inaccurate and that activists have taken information and data that is full of holes and run with it, in what has come to be known as 'global warming alarmism.' Her website includes a slew of information, including a critique of Al Gore's famous documentary, An Inconvenient Truth.
Now, as much as I appreciate scientific debate, I find the climate change skeptics to be boorish (use of this term is my own personal jeer at the recent comments of Peggy Noonan). And to explain why, I'll start by explaining my thoughts on climate change activists. I'm not an expert on climate change, so I'm not going to address the scientific talking points, but I do fully understand what climate change activists are asking of the public and our society. They are, in essence, asking us to become more responsible members of our planet; to consider the harm we do to our environment as well as to our own health; to think of the impact we will have on our children, and our children's children; to remember that we are not the only species on the Earth struggling to survive. At the root of all of this, they are really asking only one thing: for all of us to consider the consequences of our actions.
If these are the things that climate change activists want, then it's safe to conclude that the anti-alarmists and skeptics would like us to do the exact opposite; to be irresponsible and self-serving; to think nothing of the environment and human health; to let future generations fend for themselves; to utilize the Earth's organisms as a resource that can be depleted, much like our precious fossil fuels, without worry. Basically, I can only conclude that they would like us to act before thinking, addressing major problems only after they are beyond the point of correction, all in order to destroy the Earth, us along with it, leaving behind a smoldering, lifeless chunk of rock to circle the Sun.
Dramatic? Of course. True? You decide. But for as much flack as Al Gore, for example, receives for his viewpoints, it doesn't really matter if the scientific data that he brandishes like a weapon is true or not. Because at the end of the day, he's really only using it for one purpose, and that's to make us better people. The alarmists may be off-putting, but that's because it's always off-putting to hear someone tell you that you are acting like an inconsiderate and thoughtless cretin. So instead of arguing over whether the data is accurate and who's right, why don't we leave that up to the experts, and the rest of us roll up our sleeves and get down to becoming more responsible and humane people. You know...be, like, better.
Now, as much as I appreciate scientific debate, I find the climate change skeptics to be boorish (use of this term is my own personal jeer at the recent comments of Peggy Noonan). And to explain why, I'll start by explaining my thoughts on climate change activists. I'm not an expert on climate change, so I'm not going to address the scientific talking points, but I do fully understand what climate change activists are asking of the public and our society. They are, in essence, asking us to become more responsible members of our planet; to consider the harm we do to our environment as well as to our own health; to think of the impact we will have on our children, and our children's children; to remember that we are not the only species on the Earth struggling to survive. At the root of all of this, they are really asking only one thing: for all of us to consider the consequences of our actions.
If these are the things that climate change activists want, then it's safe to conclude that the anti-alarmists and skeptics would like us to do the exact opposite; to be irresponsible and self-serving; to think nothing of the environment and human health; to let future generations fend for themselves; to utilize the Earth's organisms as a resource that can be depleted, much like our precious fossil fuels, without worry. Basically, I can only conclude that they would like us to act before thinking, addressing major problems only after they are beyond the point of correction, all in order to destroy the Earth, us along with it, leaving behind a smoldering, lifeless chunk of rock to circle the Sun.
Dramatic? Of course. True? You decide. But for as much flack as Al Gore, for example, receives for his viewpoints, it doesn't really matter if the scientific data that he brandishes like a weapon is true or not. Because at the end of the day, he's really only using it for one purpose, and that's to make us better people. The alarmists may be off-putting, but that's because it's always off-putting to hear someone tell you that you are acting like an inconsiderate and thoughtless cretin. So instead of arguing over whether the data is accurate and who's right, why don't we leave that up to the experts, and the rest of us roll up our sleeves and get down to becoming more responsible and humane people. You know...be, like, better.
Monday, September 21, 2009
That's Great, But What Do We DO With It?
While enjoying my coffee and perusing through Nature this fine morning, my mind sort of went off on a tangent on what has become a bit of a pet peeve of mine. Let's start from the beginning...
A couple of years ago, I was listening to an episode of Talk With the Nation: Science Friday in which Ira Flatow, with whom I am more than a little endeared, was speaking with one of the folks from CERN about the construction and future studies of the Large Hadron Collider (LHC). It was a great interview and it left me feeling very excited about the LHC coming online, even though it wasn't scheduled to happen for many months. As always, Ira took phone calls from listeners. One caller asked something along the lines of, 'that sounds great, but what's the potential application for this?' The physicist from CERN was polite (much moreso than I would've been) in explaining that the LHC was a device built for discovery; for basic science, not applied science. The information gathered from the LHC's experiments had no direct application, but unforeseen applications and benefits could arise as a result. Oh yeah, and don't forget about uncovering the mysteries of the universe. That important too, I guess.
In general, the question 'what are the applications for this?' roughly translates into 'how will this benefit me?' I wanted the CERN physicist to say, "What do you mean applications? Did you not hear what I just said? I said Higgs boson, you fool. We're illuminating the inner workings of the universe and adding to the knowledge of the human race, not developing a new microwave oven." I know that's a little harsh, but sometimes the truth hurts.
I don't even do basic science. I do applied science, so I know that question shouldn't bother me, but it does. Why does everything have to have a direct application? Why does it always have to benefit everyone (and benefit them right away)? When did science become less about learning and discovery and all about making Average Joe's life a little easier? I know that applied science and engineering have their places in the world of science because I am, after all, an engineer, but I still feel a sense of wonder and excitement when I read about new scientific discoveries, or watch an episode of Nova, or see pictures of the LHC's massive detectors.
I just hope I'm not the only one.
A couple of years ago, I was listening to an episode of Talk With the Nation: Science Friday in which Ira Flatow, with whom I am more than a little endeared, was speaking with one of the folks from CERN about the construction and future studies of the Large Hadron Collider (LHC). It was a great interview and it left me feeling very excited about the LHC coming online, even though it wasn't scheduled to happen for many months. As always, Ira took phone calls from listeners. One caller asked something along the lines of, 'that sounds great, but what's the potential application for this?' The physicist from CERN was polite (much moreso than I would've been) in explaining that the LHC was a device built for discovery; for basic science, not applied science. The information gathered from the LHC's experiments had no direct application, but unforeseen applications and benefits could arise as a result. Oh yeah, and don't forget about uncovering the mysteries of the universe. That important too, I guess.
In general, the question 'what are the applications for this?' roughly translates into 'how will this benefit me?' I wanted the CERN physicist to say, "What do you mean applications? Did you not hear what I just said? I said Higgs boson, you fool. We're illuminating the inner workings of the universe and adding to the knowledge of the human race, not developing a new microwave oven." I know that's a little harsh, but sometimes the truth hurts.
I don't even do basic science. I do applied science, so I know that question shouldn't bother me, but it does. Why does everything have to have a direct application? Why does it always have to benefit everyone (and benefit them right away)? When did science become less about learning and discovery and all about making Average Joe's life a little easier? I know that applied science and engineering have their places in the world of science because I am, after all, an engineer, but I still feel a sense of wonder and excitement when I read about new scientific discoveries, or watch an episode of Nova, or see pictures of the LHC's massive detectors.
I just hope I'm not the only one.
Monday, September 14, 2009
Let the Sun Shine In
I read an interesting article today in Science Magazine on a potential new solar cell design that could greatly enhance energy efficiency capture from sunlight. The method utilizes carbon nanotubes (CNTs) grown between two tiny electrodes, rather than large and expensive silicon wafers, to create the photovoltaic cell. When light strikes the silicon semiconductor in a traditional solar cell, one exciton is produced, which then moves towards an electrode creating a current. Any additional energy from the photon's absorption is given off in the form of heat. In the new CNT-based cells, studies indicate that the nanotubes are able to produce more than one exciton from a single photon absorption event, increasing the overall efficiency of the cell.
Sounds great, right? So what's the catch? The catch is that this phenomenon has only been observed so far at 60 degrees Kelvin, or around -213 degrees Celcius. But that's not all. Work so far has focused on characterizing the effect in a single cell. In order for this process to be applicable to, say, heating your home, the structure would have to be multiplied many billion times in an array. And on top of that, the production of the device would have to be scaled up to make it competitive with current solar panel production.
This is all very interesting and I found the article fascinating, but it reminded me of another solar cell innovation that was awarded as one of TIME Magazine's Best Inventions of 2008. The technology, which has matured to create the west coast-based company Nanosolar, is founded on the ability to 'screen print' the solar cell. Instead of using silicon wafers, Nanosolar uses semiconductor nanoparticles dispersed in a sort of ink, which is then printed onto a substrate very similar to aluminum foil. The result is an extremely thin, very lightweight, inexpensive solar panel. And when I say very lightweight, I mean VERY lightweight: check out the TIME article to see Nanosolar CEO Martin Roscheisen with a massive solar panel perched neatly on his shoulder. Currently, Nanosolar is targeting integration of the panels into energy grids in the form of solar 'power plants,' but the rest of us non-utility folk can sign up to receive updates on when their solar panels will become available to homeowners. I, for one, may be putting my name on that list.
Sounds great, right? So what's the catch? The catch is that this phenomenon has only been observed so far at 60 degrees Kelvin, or around -213 degrees Celcius. But that's not all. Work so far has focused on characterizing the effect in a single cell. In order for this process to be applicable to, say, heating your home, the structure would have to be multiplied many billion times in an array. And on top of that, the production of the device would have to be scaled up to make it competitive with current solar panel production.
This is all very interesting and I found the article fascinating, but it reminded me of another solar cell innovation that was awarded as one of TIME Magazine's Best Inventions of 2008. The technology, which has matured to create the west coast-based company Nanosolar, is founded on the ability to 'screen print' the solar cell. Instead of using silicon wafers, Nanosolar uses semiconductor nanoparticles dispersed in a sort of ink, which is then printed onto a substrate very similar to aluminum foil. The result is an extremely thin, very lightweight, inexpensive solar panel. And when I say very lightweight, I mean VERY lightweight: check out the TIME article to see Nanosolar CEO Martin Roscheisen with a massive solar panel perched neatly on his shoulder. Currently, Nanosolar is targeting integration of the panels into energy grids in the form of solar 'power plants,' but the rest of us non-utility folk can sign up to receive updates on when their solar panels will become available to homeowners. I, for one, may be putting my name on that list.
Tuesday, September 8, 2009
The Rat King
A company from our own backyard, St. Louis-based Sigma-Aldrich, is about to utilize a brigade of genetically modified rats to wage war against a dominant and more numerous army of genetically modified mice. Seriously...
For years, mice have reigned over the world of mammal models of genetic modification. That is, it's comparatively easy to create knock-out mice with precisely controlled genetic traits using embryonic stem cells. However, rats are better human models of many diseases than are mice. So Sigma-Aldrich, with the help of the Medical College of Wisconsin, developed a method of targeting specific genes for removal using a zinc finger nuclease. Rather than using embryonic stem cells, which can be finicky to say the least, the DNA nuclease is introduced to the fertilized rat egg, where it clips particular sequences from the genome. And Voila! You've got yourself a knock-out rat. Sigma plans to have off-the-shelf rats with modifications that are popular among current researchers, as well as custom, made-to-order rats.
If business takes off, Sigma-Aldrich, who already has a strangle-hold on the laboratory chemical reagent market, could become a major player in the laboratory animal trade. This could be a great asset to the economy of St. Louis and the State of Missouri. The area is already transforming, step-by-step, into a legitimate biotechnology hub, but the Sigma Advanced Genetic Engineering Lab with their legions of genetically modified rats could be more like a running charge, complete with screams of nonsensical drivel, to position St. Louis at the forefront of the biotech industry.
For years, mice have reigned over the world of mammal models of genetic modification. That is, it's comparatively easy to create knock-out mice with precisely controlled genetic traits using embryonic stem cells. However, rats are better human models of many diseases than are mice. So Sigma-Aldrich, with the help of the Medical College of Wisconsin, developed a method of targeting specific genes for removal using a zinc finger nuclease. Rather than using embryonic stem cells, which can be finicky to say the least, the DNA nuclease is introduced to the fertilized rat egg, where it clips particular sequences from the genome. And Voila! You've got yourself a knock-out rat. Sigma plans to have off-the-shelf rats with modifications that are popular among current researchers, as well as custom, made-to-order rats.
If business takes off, Sigma-Aldrich, who already has a strangle-hold on the laboratory chemical reagent market, could become a major player in the laboratory animal trade. This could be a great asset to the economy of St. Louis and the State of Missouri. The area is already transforming, step-by-step, into a legitimate biotechnology hub, but the Sigma Advanced Genetic Engineering Lab with their legions of genetically modified rats could be more like a running charge, complete with screams of nonsensical drivel, to position St. Louis at the forefront of the biotech industry.
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