By injecting the virus directly into the spinal cord, Aebischer and Raoul aimed their weapon at the affected area. This is among the first studies in which RNA interference has been used as a therapy against a neurodegenerative disease, Aebischer says.
"We targeted the cause of the disease by knocking down mutant SOD1 in the spinal cord," Raoul says. "This helped delay the first signs of motor impairment and slowed disease progression."
Because the normal copy of the SOD1 gene performs important duties in cells, the researchers built a normal copy of the gene into the virus they delivered to the cells. This feature--removing a harmful version of a gene while replacing it with a normal one--may allow a similar technique to be used in many other diseases affected by other genes.
"This work opens a therapeutic avenue to neurodegenerative disorders including Parkinson's or Huntington's disease," says Aebischer.
In another study, Erik Storkebaum and Diether Lambrechts, PhD--working in the laboratory of Peter Carmeliet, MD, PhD, at the Catholic University of Leuven in Belgium--rescued the motor neurons attacked by ALS by providing them with a key support agent called vascular endothelial growth factor (VEGF).
VEGF was first discovered as an agent that helps grow blood vessels. It has more recently been shown to support neurons. In both rodents and people, a low level of VEGF may be a risk factor for ALS . So Storkebaum and his colleagues tested whether raising VEGF levels could better protect against ALS in a mouse model.
The investigators first delivered VEGF to the brains of ALS rats with a surgically implanted needle. The compound was added to the cerebrospinal fluid, which constantly "washes" the entire brain and spinal cord.
"When VEGF was administered one month before the disease symptoms showed u
'"/>
Contact: Leah Arinello
dawn@sfn.org
202-462-6688
Society for Neuroscience
24-Oct-2004