Problems With RNA Interference Gene Therapy
Disappointing news on the gene therapy front. RNA interference (RNAi) was once thought to have great potential for directly silencing genes and stopping them from producing functional protein products. Stanford scientists report that in a study of hepatitis B in which they attemped to silence the virus’s genes, RNAi cured some mice, caused liver poisoning in others, and death in a few.
Not everyone is panicking, though. Phillip Zamore, co-founder of Alnylam Pharmaceuticals which develops RNAi drugs, says the Stanford study followed an outdated protocol and used a higher than therapeutically necessary dose.
Dr. Mark A. Kay:
The good news about RNAi is that it uses the cell’s machinery to do its work, and that is why it’s so effective. The bad news about RNAi is that it uses the cell’s machinery to do its work. If you overload the system, you hijack the machinery from performing its normal duties.
RNAi still seems like a promising technique but obviously a lot of tweaking needs to be done before it becomes a commonly prescribed treatment.
New York Times, May 25, 2006
Technorati Tags: RNA interference, RNAi, genetics, genes, dna, disease, health
