RNA Interference as a New Tool in Therapeutics

Mirela Matokanović* and Karmela Barišić

Department of Medical Biochemistry and Haematology, Faculty of Pharmacy and Biochemistry, University of Zagreb, A. Kovačića 1, HR-10000 Zagreb, Croatia

Article history:

Received November 3, 2008
Accepted May 5, 2009

Key words:

RNA interference, small interfering RNA, off-target effects, therapeutics


The basic principle of RNA interference, a possible new therapeutic tool, involves destruction of messenger RNA upon interaction with homologous double-stranded RNA present in the cell cytoplasm. Studies have shown that both viral and non-viral small interfering RNA delivery methods and delivery of chemically synthesized small interfering RNAs to the cell can provide selective gene suppression through this mechanism, both in vitro and in vivo. Before becoming a functional therapeutic tool, there are a number of problems concerning RNA interference that should be solved. Major problems involve off-target effects, insertional mutagenesis and malignant transformation, as well as problems of delivery methods and reduction of toxicity.


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