RNAi Technology Silences Gene that Causes ALS

Scientists have used RNAi (RNA interference) in a mouse model to silence the mutant SOD1 gene, which causes familial amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's diseases. The accomplishment has high potential for treating previously untreatable diseases.

RNAi is used to shut down or silence disease-causing genes within a living cell. A research team at the University of Massachusetts Medical School (UMMS, Worcester, USA) led by Zuoshang Xu, M.D., Ph.D., designed a strategy to selectively silence the mutant SOD1 gene. By administering RNAi into the tail vein of a mouse, Dr. Xu's team demonstrated that the disease gene can be silenced. Dr. Xu has been collaborating with CytRx Corp. (Los Angeles, CA, USA), which has an exclusive license for the RNAi technology from UMMS.

"What Dr. Xu has achieved in his lab has never before been accomplished--never in the history of ALS has anyone succeeded in finding a way to attack the cause and arrest the development of the disease instead of just treating the symptoms,” said Dr. Louis J. Ignarro, Nobel Laureate and chief scientific spokesman for CytRx.

ALS is a neurodegenerative disease that causes motor neuron degeneration, skeletal muscle atrophy, and paralysis. The disease is progressive and invariably fatal.





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