Sickle cell disease corrected in human models using stem cell-based gene therapy

An exciting development. Somewhat "old news" to me and perhaps many of you of, but it's been sitting in my files for 10 months waiting to be posted. Of particular interest to me is that RNA interference (RNAi) is used in this study. While not as glamorous or as well known as stem cell therapy, RNA interference is much further advanced and has significant short term potential. the concept also raises an interesting analogy with traditional chinese medicine, as RNAi is a way to eliminate the pathological by-products caused by poorly functioning organs. (In TCM, poor organ function is said to result in toxic accumulation that is the proximate cause of all disease.)

In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology.