Monkeys and RNAi (mostly RNAi)
RNAi (short for ribonucleic acid interference) is a technique that is used to silence gene expression in a variety of animals. Researchers recently reported in Nature that they had successfully used RNAi to drastically reduce LDL (i.e. bad) cholesterol in monkeys.
Simply put, RNAi works by introducing a small piece of RNA (siRNA - small interfering RNA) that is similar to a specific messenger RNA (mRNA - RNAs that are used to generate proteins) into a cell. Specific cellular machinery then uses this small piece of RNA to destroy all similar mRNAs. This has the effect of keeping any new proteins of the type targeted by the siRNA from being made. As such, with this technology, you can disrupt any functions that require the protein that you target with RNAi.
The researchers were able to target liver cells with siRNAs designed to silence apolipoprotein B (a protein component of LDL cholesterol). This had the effect of reducing LDL cholesterol by 80%. Not bad.
The next step is to test RNAi in humans to determine if it is a viable treatment. If it is, the potential applications could be staggering - just reducing LDL by 80% would eliminate countless cases of cardiovascular disease. In addition, there are many diseases that could be effectively treated by shutting off one or two proteins.
As far as safety goes, RNAi was well tolerated in the test monkeys. However, if an siRNA shuts down the function of the wrong protein(s) there could be serious problems. Another source of side effects could come from the method of siRNA administration or cell targeting.
All in all, this could be a huge step forward in treating genetic and other diseases.
Category: Science
Simply put, RNAi works by introducing a small piece of RNA (siRNA - small interfering RNA) that is similar to a specific messenger RNA (mRNA - RNAs that are used to generate proteins) into a cell. Specific cellular machinery then uses this small piece of RNA to destroy all similar mRNAs. This has the effect of keeping any new proteins of the type targeted by the siRNA from being made. As such, with this technology, you can disrupt any functions that require the protein that you target with RNAi.
The researchers were able to target liver cells with siRNAs designed to silence apolipoprotein B (a protein component of LDL cholesterol). This had the effect of reducing LDL cholesterol by 80%. Not bad.
The next step is to test RNAi in humans to determine if it is a viable treatment. If it is, the potential applications could be staggering - just reducing LDL by 80% would eliminate countless cases of cardiovascular disease. In addition, there are many diseases that could be effectively treated by shutting off one or two proteins.
As far as safety goes, RNAi was well tolerated in the test monkeys. However, if an siRNA shuts down the function of the wrong protein(s) there could be serious problems. Another source of side effects could come from the method of siRNA administration or cell targeting.
All in all, this could be a huge step forward in treating genetic and other diseases.
Category: Science
posted by Tokatakiya at 5:03 PM
0 Comments:
Post a Comment
<< Home