Gene silencing made practical: siRNA used to treat neurogenerative disorders in the lab

Darkfrog submitted, created time 5 months 4 weeks (www.jbc.org)

This abstract is about as easy to read as sixteenth-century tax law, but here's the gist: Human neurogenerative disorders, like Huntington's, Parkinson's, Alzheimer's and amyotrophic lateral sclerosis (Lou Gherig's disease) are all caused or exacerbated by the expression of mutant genes. If we switch off the genes--or at least keep them from producing proteins--then the patients might get better. One way to switch off a gene is to inject the cell with a siRNA (short interfering RNA) that complements the gene's product mRNA. This is called gene silencing. The gene is still there, but it doesn't affect anything any more.

However, injecting raw siRNA into a full-suzed human with blood, cell walls and an immune system doesn't always work. Like most other raw chemicals, the siRNA would be broken down before it could do any good. These researchers used a chemically stabled version of siRNA on lab animals. Guess what happened?

Keywords: gene silencing Huntingtons disease Alzheimers disease Parkinsons alzheimers huntingtons amyotrophic lateral sclerosis small interfering RNA (siRNA) lou gherig's disease ALS