Gene Therapy Restores Sight to Blind Mice and Increases Heart Attack Survival from 60 to 80% in Mice

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1. The researchers used a specially engineered virus to deliver extra copies of the gene for Nerve growth factor (NGF) into the hearts of mice. Thirty days after having a heart attack, 80 per cent of the mice that received this treatment were still alive, compared with only 60 per cent of mice that did not. The pumping ability of the heart was also significantly improved in mice that received the NGF gene.

In contrast, mice that were given an antibody that neutralised NGF fared significantly worse after they had a heart attack, with higher rates of cell death and more severely impaired heart function.

When the researchers looked at post mortem human hearts that had suffered a heart attack, they found that levels of NGF near the blood-starved part of the heart were higher than normal, suggesting that increasing NGF production is a normal part of the tissue’s response to oxygen deprivation.

Dr Emanueli, Reader of Research in Experimental Cardiovascular Medicine Research in the Bristol Heart Institute (BHI) at the University of Bristol, said: “We’ve shown that in mice, NGF gene therapy improves survival rates after a heart attack, through both a direct protective effect on heart muscle cells and by stimulating the growth of new blood vessels.

“In the laboratory, we can deliver therapy directly to the heart at the time of the heart attack. But in a clinical context the therapy will have to be given hours or days later, ideally without the need for surgery. So next we need to see if we can get similar results in this situation.

“I’m convinced that NGF has huge potential for treating heart attack patients.”

2. Scientists from Buffalo, Cleveland, and Oklahoma City made a huge step toward making the blind see, and they did it by using a form of gene therapy that does not involve the use of modified viruses.

Scientists described how they used a non-viral, synthetic nanoparticle carrier to improve and save the sight of mice with retinitis pigmentosa, an inherited disease characterized by progressive vision loss and eventual blindness.

“We hope the results of our study will be instrumental in generating a cure for the debilitating blindness associated with retinitis pigmentosa and other inherited and acquired retinal diseases,” said Muna I. Naash, Ph.D., a researcher involved in the work from the Department of Cell Biology at the University of Oklahoma Health Sciences Center in Oklahoma City. “Compacted DNA nanoparticles are an exciting treatment strategy for these diseases and we look forward to exciting new developments.”

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