Washington: Scientists from California Institute of Technology (Caltech) have suggested a gene therapy that is capable of stalling the development of Huntington`s disease.
They found that one of the two intrabodies had some negative results, actually increasing Huntington`s-related mortality in one model. But the other intrabody- called Happ1- was an unqualified success, restoring motor and cognitive function to the mice, and reducing neuron loss as well as toxic protein accumulation. And in one model, it increased both body weight and life span. "Our studies show that the use of intrabodies can block the parts of mutant huntingtin that cause its toxicity without affecting the wildtype, or normal, huntingtin—or any other proteins," said Patterson. In other words, he says, this has the potential to be the kind of "silver-bullet therapy" that many medical researchers look for. Despite the fact that this disorder has a single-gene origin, current treatments tend to address the symptoms of the disease, not its cause. That means it is currently impossible to prevent the disease from doing significant damage in the first place. The study appears in the Journal of Neuroscience. ANI
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