Here is an interesting news article about the use of RNAi in human diseases:
RNAi trailblazer Alnylam has published a paper in the journal Human Molecular Genetics that details RNAi's ability to silence a gene implicated in early onset torsion dystonia. Dystonia is a neurological movement disorder in which sustained muscle contractions cause twisting and repetitive movements or abnormal posture. According to Dinah Sah, Ph.D., Senior Director of Research at Alnylam, the data demonstrates RNAi therapeutics can discriminate between a disease target of interest and a closely related gene, which could help treat genetic disorders.
"We are encouraged by these results, as well as results we have seen to date with our other neurological RNAi therapeutic programs, such as Huntington's disease, Parkinson's disease, and others," added Sah.