Small Silencing RNAs and Gene Therapy
Authors: Grimm D
CellNetworks People: Grimm Dirk
Journal: ENCYCLOPEDIA OF LIFE SCIENCES, John Wiley & Sons, Ltd: Chichester. doi: 10.1002/9780470015902.a0022396

Within one decade, RNA interference (RNAi), i.e., the sequence-specific knockdown of gene expression triggered by small silencing RNAs, has rapidly matured from a biological curiosity into our single most promising biotherapeutic for a wide array of human diseases. Its exciting looming clinical translation is particularly accelerated by the increasingly pursued juxtaposition of RNAi technologies with established gene therapy methodologies. A key player fostering this mutual attraction of two potent clinical modalities are viral gene transfer vectors, whose abilities to mediate stable, efficient and tailored transduction of genetic material into recipient cells pave the way to fully harness the power of RNAi therapies. Adding to the enormous promise of combining small silencing RNAs and gene therapy are latest findings on the role of endogenous micro-RNAs for various human diseases, further enlarging our already fertile chest of tools and targets for intervention and fortifying the optimism that RNAi gene therapies will soon become a clinical reality.