Tilly G, Chapuis J, Vilette D, Laude H, Vilotte J L
Laboratoire de Génétique Biochimique et de Cytogénétique, Institut National de la Recherche Agronomique, 78352 Jouy-en-Josas Cedex, France.
Biochem Biophys Res Commun. 2003 Jun 6;305(3):548-51. doi: 10.1016/s0006-291x(03)00805-2.
Prion diseases are fatal neurodegenerative disorders associated with an abnormal isoform of the PrPc host-encoded protein. Invalidation of the Prnp gene, that encodes PrPc, led to transgenic mice that are viable, apparently healthy, and resistant to challenge by the infectious agent. These results indicated that a down-regulation of the Prnp gene expression is a potential therapeutic approach. In the present report, we demonstrate that RNAi targeted towards the Prnp mRNA can efficiently and highly specifically reduce the level of PrPc in transfected cells. It, thus, indicates that RNAi is an attractive therapeutic approach to fight against prion diseases.
