A team of researchers from the University of Missouri, led by FightSMA Science Director Chris Lorson, is making strides in increasing the lifespan of mice with SMA. Here’s a quote from an article published by the University of Missouri:
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.”
Lorson says this treatment method is very close to becoming a reality for human patients. Jacqueline Glascock, co-author of this study, was a presenting researcher at the FightSMA 2012 Annual Research Conference. A study on the research was published in Biochemical and Biophysical Research Communications.
Were there also signs that this approach reverses some of the effects of SMA?
Did the mice that were treated with gene therapy gained some strenght?