Scientists using gene therapy to stall Huntington's disease
1 Nov 2009, 1021 Hrs

Add comment          Mail          Print          Write to Editor

Washington, Nov 1 Scientists from California Institute of Technology (Caltech) have suggested a gene therapy that is capable of stalling the development of Huntington's disease.

Huntington's disease is a neurodegenerative disorder with a genetic basis. The disorder has its roots in a mutation in a protein called huntingtin, or Htt. (The gene itself is also referred to as the huntingtin gene.)

"Gene therapy in these models successfully attenuated the symptoms of Huntington's disease and increased life span," said Paul Patterson, the Anne P. and Benjamin F. Biaggini Professor of Biological Sciences.

The researchers began to look at the efficacy of two different intrabodies that had been shown, in cell cultures and fruit-fly models, to reduce the accumulation of toxic Htt protein.

To see whether those effects would hold true in mammalian systems as well, the team tested the intrabodies in a series of five different mouse models of Huntington's.

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)

Related Latest

New discovery could make plants disease resistant and high-yielding Radiation therapy can boost longevity in rare pancreatic cancer patients Coimbatore farmer gets high yield of tomatoes, using green house technology Laser therapy can worsen skin cancer Pakistan using terror as instrument of state policy: PM Laser therapy worsens skin cancer: Study Sleep apnea ups heart disease risk in kidney transplant patients New therapy offers hope for mental disorder patients Heart disease found in 3,000 year old Egyptian mummies Obesity epidemic negating efforts to cut heart disease risks

Related Latest

Warnie says 'Pup' Clarke is his new bunny in 20-20 match Assam bomb blast death toll rises to seven (Update Assam blast) Windies won't wilt against Aussies, says coach Williams Central Excise, Customs official protest in Delhi for better pay Teenager charged for mutilation murder of Brit Indian Orissa Govt orders heavy security deployment in Korapat District Ferguson's overhaul plan puts greats under scrutiny Arrest warrant issued against two ministers in Karzai's cabinet Former Australian cricket stars question Clarke's capacity to captain Headley always felt pulled between East and West: Investigators

• Windies won't wilt against Aussies, says coach Williams
• Assam bomb blast death toll rises to seven (Update Assam blast)
• Orissa Govt orders heavy security deployment in Korapat District
• Warnie says 'Pup' Clarke is his new bunny in 20-20 match
• Central Excise, Customs official protest in Delhi for better pay

• भारतीय क्रिकेट की मजबूत दीवार
• कंगारूओं के आगे ढेर, चीतों से कैसे मुकाबला करेंगें शेर
• Reducing greenhouse gases may not be enough to slow global warming
• बीकानेर महापौर चुनावः एक विश्लेषण
• Deepawali: The Festival Of Lights