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...
Subscribe to:
Posts (Atom)
