
Gene Therapy Makes a Comeback
Maligned for many years after a 1999 study led to the death of an 18-year-old patient, gene therapy may have finally recovered its promise as breakthrough medicine. In recent years the field has taken positive steps, culminating most recently in the successful—and safe—treatment of hemophilia B.
The new treatment uses a modified cold virus, outfitted with a gene that hemophiliacs are missing. The gene in question allows the body to produce Factor IX, which helps the blood to clot.
The study, published online in December by The New England Journal of Medicine, only enrolled six patients—but after the therapy, four of them could halt their regular Factor IX infusions, and the other two could safely reduce the frequency of their infusions.
Coupled with another success earlier this year (treating children born with severe combined immune deficiency), along with new ways of delivering missing genes, the field is offering renewed hope for treating genetic disorders.—Katherine Harmon
Image of gene therapy courtesy of the Genetics Home Reference a service of the U.S. National Library of Medicine®
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12 Comments
Add CommentThe rise and fall of tides have been overlooked. A large floating body, say a manufactured island, rises and falls with the tide around a column fixed to the sea floor. A system of rack and pinion gears spin generators on the island.
Reply | Report Abuse | Link to thisOr the 25 ft. rise limit of an Archimedes pump can be overcome by staging a series every 25 ft. The pump to be driven by its stream flow acting on propellers. The pump could raise water to an effective height for hydro electric generation.
Or a mechanical sea lift can be driven by a floating push rod rising and falling with the tide. The derived reservoir of sea water could be commercially viable for fish farming,salt producing, or a large form for hydro stuff.
Interesting that they removed my comment from yesterday, which was: "None of these are really science stories in the sense of pure science. Pathetic".
Reply | Report Abuse | Link to thisDoubly pathetic that they would remove this comment.
@bigbopper
Reply | Report Abuse | Link to this"Never attribute to malice what can be attributed to incompetence"
-- some old fart's saying
So unless you received email saying that SA was deliberately removing your comment, I suggest just chalking it up to some error.
That being said, I'll agree that many of the stories are not pure science. E.g., Steve Jobs' death. On the other hand some are indeed science ... the Higgs Boson announcement and the neutrino announcement (although both of those may be too premature to be long-lasting).
Why does a Science Magazine give Steve Jobs' unfortunate death a top billing without even mentioning the passing of Dennis Ritchie, the father of the C programing language and the UNIX operating system the backbones of the digital infrastructure that made possible Jobs consumer products?
Reply | Report Abuse | Link to thisVery illustrative account of science stories in 2011.
Reply | Report Abuse | Link to thisDr.A.Jagadeesh Nellore(AP),India
E-mail: anumakonda.jagadeesh@gmail.com
I'm only surprised that Steve Jobs wasn't given credit for the "Arab Spring", as well as the fall of the Iron Curtain...
Reply | Report Abuse | Link to thisThe report of a faster than light neutrino is very interesting. The late Isaac Asimov would probably have chuckled and commented that this might be the first reported instance of a subatomic particle that he, and other science fiction authors, had dubbed the 'tachyon', or precsiely the subatomic particle that you need to investigate whether it is possible to construct a Faster-Than-Light space ship engine.
Reply | Report Abuse | Link to thisLook Out, Vulcans!
Look Out, Klingons!
Here Comes The Terrans!
I thought some Dutchman solved the FTL-neutrino problem. See http://www.forbes.com/sites/johnfarrell/2011/10/14/netherland-scientist-claims-solution-to-the-ftl-neutrino-problem/
Reply | Report Abuse | Link to thisHe is Ronald van Elburg at the University of Groningen.
For the more-technically minded, look at http://www.technologyreview.com/blog/arxiv/27260/ and http://home.kpn.nl/vanelburg30/Papers/RAJvanElburg_TimeOfFlight_Preprint.pdf
Simply put, the neutrinos' distance-of-travel was measured in one coordinate system (the earth's), and the time-of-flight was measured in a different coordinate system -- that of the GPS satellite.
Imagine a very long spaceship with clocks at the bow and the stern, traveling at a large fraction of the speed of light. The clocks are synchronized in the ship's own frame-of-reference. However, to an observer viewing the ship from the side (so that the observer is equidistant from both clocks), the bow clock will appear to be slow compared to the stern clock.
By the way, if tachyons (particles with imaginary rest masses that are obligated to travel faster than the speed of light) do emit some analogue of Cherenkov radiation, the loss of energy will make them go FASTER, not SLOWER, until they reach infinite speed (in the frame-of-reference of the matter they are traveling through), when they'd have zero dynamic mass, but will still have finite, but ambiguous, momentum (imc). This looks like an impossibility, so I conclude that tachyons do not exist.
Talk about been dumbed down! With the list of REAL Science given in the introduction I actually believed Sci-Am was going to give us a list that they were worthy of, but instead we get news items that we get on the 6 o’clock news every night.
Reply | Report Abuse | Link to thisSteve Jobs for one is not science, it is sad for his family and friends and those poor unfortunates that use Apple, but it is NOT science. I agree with lcallen3, I did not know Dennis Ritchie had died, if Jobs should on the list so should Ritchie, I studied him about thirty years ago when I studied computers and programming in particular C, then C+ and all the work he did.
The weather is news but again is not science news. The Higgs Boson, was news at the time but until it has been proven it should not be included in any top ten list. Nor should the Subluminal neutrino be included for the same reasons.
So come on Sci-Am get your act together give us a list both you and your readers can be proud of. Your followers know you are capable, you have done so in the past, it may have been some time in the past but you have done it!
BTW, has anyone else had a problem signing in, every time I sign in I have to reset my password, and I know I have the right password as I wrote it down when I joined up, but Sci-Am just seems to keep messing me about, does it happen to any anyone else?
At Christmas 2011, from womb of Quantum Biophysical Semeiotics (www.semeioticabiofisica.it) has born a new clinical diagnostic method, Brain Sensor Bedside Evaluation, which open a new road in the field of physical Semeiotics:http://blogs.nature.com/spoonful/2011/12/hpv-vaccination-for-boys-called-into-question.html#comment-574 .
Reply | Report Abuse | Link to thisAlas, because SciAm is no longer a "Science Magazine".
Reply | Report Abuse | Link to thisDear Editors, Notoriously, type 2 DM is a growin epidemic. Aiming to find new road in the war against ist, I like to emphasise that Adiponectin influences the body's response to insulin, increasing insulin receptors sensitivity, as well as insulin secretion. In addition, Adiponectin blood Level is significantly reduced (1). An untill now Adiponectin interesting action mechanism is the stimulation of insulin-independent way of glucose absorption at intestinal, renal, skeletric muscle level. In healty individuals, bedside stimulation of Adiponectin secretion, I have illustrated in a previous paper(1), brings about simultaneuosly maximal microcirculatory activation, type I, associated, in above-mentioned biological systems, indicating increased work in related parenchymas, according to Angiobiopathy theory (3).
Reply | Report Abuse | Link to thisOn the contrary, in all FIVE stages of type 2 Diabetes Mellitus, such a microcirculatory activation is not "simultaneous", but shows different degree of latency time in sec, as well of microcirculatoy activation, paralleling the seriousness of underlying disorder, minimal in Diabetic Constitution(2). As a consequence, I suggest this clinical method in detecting type 2 DM on very large scale, starting from the first stage, i.e., diabetic Constitution.
References.
1) Biophysical-Semeiotic bed-side Evaluation of Adiponectin in classic and variant Pre-Metabolic and Metabolic Syndrome. http://www.semeioticabiofisica.it/semeioticabiofisica/Documenti/Eng/Adiponectin%20lavoro%20engl.doc
2) Stagnaro S., West PJ., Hu FB., Manson JE., Willett WC. Diet and Risk of Type 2 Diabetes. N Engl J Med. 2002 Jan 24;346(4):297-298.(MEDLINE)
3) Sergio Stagnaro. Quantum biophysical semeiotics. NeuroQuantology | September 2011 | Vol 9 | Issue 3 | Page 459‐467. http://www.neuroquantology.com/index.php/journal/issue/current/showToc
4) Stagnaro Sergio. Epidemiological evidence for the non-random clustering of the components of the metabolic syndrome: multicentre study of the Mediterranean Group for the Study of Diabetes. Eur J Clin Nutr. 2007 Feb 7; [MEDLINE]