Research Topics
Our Goals
RNA Technologies aims to study RNA to fundamentally understand biology, and so to find druggable pockets in the molecular processes of cells. Bioinformatics and computational tools in general are already the hallmark of IIT’s research on RNA. Several research pipelines have been implemented and are used daily at IIT. RNA therapeutics will take advantage of the studies developed at CMP3VdA and the Joint Labs with several clinical partners as the Gaslini Children’s Hospital and the San Martino Hospital in Genoa.
Following our recent translational research on RNA and genomics, we expect to deliver: 1) knowledge about the regulatory mechanisms of RNA in the context of human disease, with a focus on neurodegenerative and neoplastic diseases; 2) generation of a robust IP portfolio in diagnostic and therapeutic applications; and 3) at least one RNA-based product ready for clinical experimentation. The ambition of the RNA Technologies Program is to drug the undruggable.
Research Topics
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The non-coding r-EvolutionOne of the most unexpected surprises of the Human Genome Project was the discovery that the number of protein-coding genes found in our genome was significantly smaller than expected.
The scientific community was thus faced with a true enigma: how is it possible to explain the incredible level of complexity and phenotypic diversity of human being starting from such a modest set of genes? The discovery of the world of non-coding RNAs revealed the answer. Thanks to new omic technologies such as microarrays and next-generation sequencing (NGS), scientists have discovered that the human genome bears many thousands of non-coding RNAs. Biology is slowly understanding that it is precisely these molecules that have allowed the development of complexity and the diversification of gene regulation that has allowed the evolution of species. |
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The non-coding r-EvolutionOne of the most unexpected surprises of the Human Genome Project was the discovery that the number of protein-coding genes found in our genome was significantly smaller than expected.
The scientific community was thus faced with a true enigma: how is it possible to explain the incredible level of complexity and phenotypic diversity of human being starting from such a modest set of genes? The discovery of the world of non-coding RNAs revealed the answer. Thanks to new omic technologies such as microarrays and next-generation sequencing (NGS), scientists have discovered that the human genome bears many thousands of non-coding RNAs. Biology is slowly understanding that it is precisely these molecules that have allowed the development of complexity and the diversification of gene regulation that has allowed the evolution of species. |
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Non-coding RNAs: one name for many speciesThe term non-coding RNA is very general and includes several classes of RNA molecules, which have in common the ability to perform a regulatory function without generating of a protein product. Classes of interest include long non-coding RNAs (lncRNAs), small non-coding RNAs (sncRNAs) and transcripts derived from Transposable Elements (TEs), such as SINE (short interspersed nuclear element) and LINE (long interspersed nuclear element). Among sncRNAs, several classes have been identified including enhancer RNA (eRNAs), microRNAs (miRNA), PIWI-interacting RNAs (piwiRNAs) and circular RNA (circRNAs).
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Non-coding RNAs: one name for many speciesThe term non-coding RNA is very general and includes several classes of RNA molecules, which have in common the ability to perform a regulatory function without generating of a protein product. Classes of interest include long non-coding RNAs (lncRNAs), small non-coding RNAs (sncRNAs) and transcripts derived from Transposable Elements (TEs), such as SINE (short interspersed nuclear element) and LINE (long interspersed nuclear element). Among sncRNAs, several classes have been identified including enhancer RNA (eRNAs), microRNAs (miRNA), PIWI-interacting RNAs (piwiRNAs) and circular RNA (circRNAs).
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Functions of Non-coding RNAs: from gene expression regulation to personalized medicinelncRNAs are exemplary of the versatility of these systems. They are flexible modular scaffolds that combine a structure with independent domains that bind protein complexes, with the recognition and modulation of RNA/DNA targets.
These classes of non-coding RNAs present virtually limitless opportunities to spatio-temporally modify gene expression; RNA-based drugs can thus extend the druggable genome with high specificity to both protein-coding genes and regulatory regions, and therefore provide an almost unlimited reservoir of new pharmacological agents. Further, they are the quintessential personalized medicine, since they can be tailored to the genomic repertory of a single patient: this is critical for a step change of therapeutic intervention, moving from a one-size-fits-all approach to personalised medicine.
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Functions of Non-coding RNAs: from gene expression regulation to personalized medicinelncRNAs are exemplary of the versatility of these systems. They are flexible modular scaffolds that combine a structure with independent domains that bind protein complexes, with the recognition and modulation of RNA/DNA targets.
These classes of non-coding RNAs present virtually limitless opportunities to spatio-temporally modify gene expression; RNA-based drugs can thus extend the druggable genome with high specificity to both protein-coding genes and regulatory regions, and therefore provide an almost unlimited reservoir of new pharmacological agents. Further, they are the quintessential personalized medicine, since they can be tailored to the genomic repertory of a single patient: this is critical for a step change of therapeutic intervention, moving from a one-size-fits-all approach to personalised medicine.
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