SMAHeadlines.com

Gene Therapy Treatment Extends Lives of Mice With Fatal Disease

A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) — the leading genetic cause of infantile death in the world.

SMA is a rare genetic disease that is inherited by one in 6,000 children who often die young because there is no cure. Children who inherit SMA are missing a gene that produces a protein which directs nerves in the spine to give commands to muscles.

The MU team, led by Christian Lorson, professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology, introduced the missing gene into mice born with SMA through two different methods: intravenously and directly into the mice’s central nervous systems. While both methods were effective in extending the lives of the mice, Lorson found that introducing the missing gene directly into the central nervous system extended the lives of the mice longer.

“Typically, mice born with SMA only live five or six days, but by introducing the missing SMN gene into the mice’s central nervous systems, we were able to extend their lives 10-25 days longer than SMA mice who go untreated,” said Lorson, who works in the MU Bond Life Sciences Center and the College of Veterinary Medicine. “While this system is still not perfect, what our study did show is that the direct administration of the missing gene into the central nervous system provides some degree of rescue and a profound extension of survival.”

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Synthetic RNA Lessens Severity of Fatal Disease

Science Daily | November 21, 2011

A team of University of Missouri researchers have found that targeting a synthetic molecule to a specific gene could help the severity of the disease Spinal Muscular Atrophy (SMA) — the leading genetic cause of infantile death in the world.

“When we introduced synthetic RNA into mice that carry the genes responsible for SMA, the disease’s severity was significantly lowered,” said Chris Lorson, researcher at the Bond Life Sciences Center and professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology. “The mice that receive synthetic RNA gain more weight, live longer, and had improvements in motor skills. These results are very exciting.”

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MU Researchers Find New Insight into Fatal Spinal Disease

HealthCanal.com | September 27, 2011

Michael Garcia, MU associate professor of biological sciences, found a communication breakdown between nerves and muscles that may provide new insight into the fatal human disease known as spinal muscular atrophy.

COLUMBIA, Mo. – Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy (SMA).

“Critical communication occurs at the point where nerves and muscles ‘talk’ to each other. When this communication between nerves and muscles is disrupted, muscles do not work properly,” said Michael Garcia, associate professor of biological sciences in the College of Arts and Science and the Bond Life Sciences Center. “In this study, we found that delivery of ‘the words’ a nerve uses to communicate with muscles was disrupted before they arrived at the nerve ending.”

This would be similar to the idea of someone opening their mouth to talk, but nothing comes out. The words are not there to come out, so no communication is transmitted to the other person.

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