New gene therapy approach could save people with muscular atrophy disease -
A discovery by the scientist Dan Rodgers Washington State University and collaborator Paul Gregorevic could save millions suffering from muscle wasting disease.
the result of the four-year project team is a therapeutic approach of the new gene. The work was published in the July 20 Science Translational Medicine , a journal of the American Association for the Advancement of Science.
"Chronic disease affects more than half of the world population," said Rodgers, a professor of animal sciences and director of the Washington Center for muscle biology. "Most of these diseases are accompanied by muscle atrophy.
" It occurs with chronic infection, muscular dystrophy, malnutrition and old age, "he said." About half of people who die of cancer are actually dying lose muscle and there is no one treatment out there who approaches.
family history inspires research for the treatment
"I have a strong motivation to do something about it, to do more than just publish results, "said Rodgers, who teamed with Gregorevic Baker IDI Heart and Diabetes Institute in Australia. "My father died of cachexia," the wasting disease caused by cancer, "and my nephew has Duchenne muscular dystrophy, an incurable and fatal disease that could claim his life in his teens.
" others have tried and failed to develop treatments for muscle wasting, "said Rodgers," and even some drugs have caused serious security problems. Our targeted approach only affects the muscles and completely avoids these problems, which is why we believe we have a solution. "
the paper's senior author Catherine Winbanks, a postdoctoral fellow Gregorevic, details how the researchers constructed muscle healthy mice and prevent the loss of skeletal and cardiac muscle in mice with tumors.
debilitating muscular effect of the hormone blocked
in cachexia, tumors secrete hormones that cause deterioration of muscles. Indeed, the body eats its own muscles, causing weakness, frailty and fatigue
"What kills a lot of people are not losing skeletal muscle, but the heart muscle," said Rodgers' heart. literally shrinks, causing heart failure "
researchers have long sought to stop this process, but failed to find a safe That's because the hormones that cause waste -. .. in particular, a natural hormone called myostatin - play important roles in the rest of the body
Rodgers and Gregorevic needed a way to stop myostatin, but only in the muscles Their solution:. . an adeno-associated virus - a virus that specifically targets Benin heart and skeletal muscle
the virus delivers a small piece of DNA - a signaling protein called Smad7. - in the muscle cells then two Smad7 signaling proteins Smad2 and Smad3 called blocks, which are activated by myostatin and other hormones from muscle atrophy. by blocking these signals, stops the fan Smad7 muscles.
"is the breaking Smad7 natural body and by inhibiting the inhibitor, you build muscle, "said Rodgers.
For patients with cachexia, such therapy could massively increase their chances of survival.
"instead of having a year to the fight against cancer, you would have 10 or 15," said Rodgers.
start work to develop commercial drugs
In 2015, Rodgers launched AAVogen, a company that will develop this discovery into a commercial drug, AVGN7.
He worked with Norman Ong, an associated license technology WSU office marketing, patent, seed funding and recruitment for AAVogen. Using funds from the allocation of funds from the WSU trade gap, the Rodgers laboratory will determine the lowest effective dose for AVGN7.
"We want to transform the WSU discoveries in the real world uses that benefit the public," said Ong. "Dan is a busy scientist, so we are proud to help and connect with AAVogen good people. "
"I formed this company with one goal: to move the science in society, to see applied," said Rodgers' office marketing WSU was instrumental and invaluable in this effort..
"Now we have a company with the potential to save many lives," he said.
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