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  New trends in chemistry and biotechnology of peptide nucleic acidS

Minerva Biotecnologica 2008 December;20(4):175-81

language: English

PNA-mediated alteration of mRNA splicing

Breveglieri G. 1, Pensato S. 3, Salvatori F. 1, Saviano M. 3, Pedone C. 3,4, Romanelli A. 4, Gambari R. 1,2

1 BioPharmaNet, Department of Biochemistry and Molecular Biology University of Ferrara, Ferrara, Italy
2 Laboratory for the Development of Pharmacological and Pharmacogenomic Therapy of Thalassaemia Biotechnology Center, University of Ferrara, Ferrara, Italy
3 Institute of Biostructure and BioimagingUniversity of Naples, Naples, Italy
4 School of Biotechnological Sciences University of Naples “Federico II”, Naples, Italy


PNAs are expected to be very active in altering the splicing pattern of target RNA sequences. Several reports have been published on this specific task, focused on analysis of PNA altering splicing and vehiculated with a variety of delivery systems. The conclusions, here reviewed, are that PNAs altering splicing have important biological effects. In our own laboratory, we have studied the in vitro effects of a 14-mer PNA targeting a portion of the b-globin gene intron 1, suggesting that this strategy might be of interest to counteract the excess production of b-globin. Furthermore, the data obtained on the same 14-mer PNA, conjugated to the SV40 NLS peptide (b-globin-PNA/NLS) to enhance the PNA concentration in cells, demonstrate that the NLS peptide does not interfere with inhibition of the splicing.

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