Courtesy of John Rossi
Citing concerns over efficacy, stability, and specificity, many researchers develop localized RNAi therapeutics strategies, such as for use in the eye. A novel variantion on this approach is being developed in which a patient's blood stem cells are transfected with a lentiviral vector expressing an anti-HIV siRNA. Those cells are then reintroduced to the patient, where the hope is that the cells will propagate and develop into mature blood cells capable of fending off HIV infection.
Traditional gene therapy is built on a simple premise: If the absence of a gene product causes disease, then adding the missing gene will cure it. But recently some researchers have turned that idea upside down, using gene therapy to silence genes gone bad. The approach takes advantage of a technique called RNA interference (RNAi) to specifically destroy a targeted mRNA and thereby eliminate the resulting protein.
An RNA-dependent, posttranscriptional ...
