Università Degli Studi Di Roma "La Sapienza"

Consiglio Nazionale Delle Ricerche

Irene Bozzoni

Alessandro Rosa

Michela Alessandra Denti

Davide Cacchiarelli

Julie Martone

Elisa Caffarelli

Tania Incitti

Pietro Laneve

Fernanda Gabriella De Angelis

Erika Girardi

Alessandro Fatica

The invention relates to the therapy of Duchenne Muscular Dystrophy (DMD) by means of modulating the amount of a specific miRNA (miR-31).

miR-31 in duchenne muscular dystrophy therapy

The authors describe an expression system for the correct, stable and effective expression in mammalian cells of a siRNA comprising: a) a polymerase II RNA-dependent promoter derived from the U1 RNA gene; downstream from the sequence b) suitable restriction sites for the cloning of the sequence transcribing the pre-siRNA; downstream from these sites c) sequences derived from the sequences at 3' of the gene for U1 snRNA that are necessary and sufficient for the correct formation of 3&#x2032; of the presiRNA.

siRNA expression system

Cloning and characterization of the gene for Mndependent RNA-sc endoribonucleasic activity able to generate 2'-3&#x2032; cyclic phosphate and 5&#x2032;OH ends.

Purification, cloning and biochemical characterization of XendoU, endoribonucleasic activity involved in small nuclear RNA splicing-independent biosynthesis in Xenopus laevis

The invention is drawn to a chimeric ribozyme molecule that comprises a U1 snRNA component and a ribozyme component where the chimeric ribozyme targets an mRNA in the spliceosome in the nuclease of a cell.

Ribozyme-snRNA chimeric molecules having a catalytic activity for

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Rome, Italy

Irene Bozzoni