WP 6.2
Development of RNA-based and RNA-targeting compounds for therapeutic uses

General objectives

Design, synthesis, selection and characterization of compounds with ‘beyond-state-of-the art’ properties in terms of selectivity, in vivo resistance and reduced immune response, binding strength to the target, potentialities of control by external, eg light, or cellular stimuli. Optimization of a platform for mRNA therapeutics.

Methodology

Advanced Organic Synthesis, Selex, Optical Spectroscopies, High-performance Computing. Structural and Molecular Biology, Nanodelivery, Nanosensing, RNA Biology, High-throughput screening.

Task 6.2.1

Task 6.2.2

Task 6.2.3

Task 6.2.1

Task Leader: Claudia Sissi
PI: Claudia Sissi, Michele Saviano, Walter Cabri

PI: Walter Cabri

We aim to overcome some RNA drug limitations by implementing new platforms based on a multi-targeted (multi-pronged) approach that includes different modalities that are now under scrutiny essentially in the USA by a few companies and research groups. The development of new chemical entities (NCE) based on different modalities can...

PI: Michele Saviano

Peptide Nucleic Acids are one of the best-performing chemical tools for DNA/RNA recognition and manipulation. PNA properties can be changed using chemical modification, thus regulating affinity and selectivity for DNA, inserting reporter groups, regulating cellular uptake. The combination of PNAs with nanostructured materials can lead to several important applications, not...

PI: Claudia Sissi

MELANOMA is the most lethal form of cutaneous cancer and the fifth most frequently diagnosed cancer. Emerging evidence support a functional role of various long-non coding RNAs (lncRNAs) in melanoma tumorigenesis according to different specific patterns. With the aim to suppress cell proliferation, we will develop unprecedentedly modified antisense oligonucleotides...

Task 6.2.2

Task Leader: Roberto Improta
PI: Roberto Improta, Momina Condorelli, Laura Cerchia, Massimo Olivucci, Francesco Ricci

 

PI: Francesco Ricci

Development and optimization of synthetic DNA/RNA nanoscaffolds and nanostructures for drug delivery and imaging purposes. We will exploit the “designability” of nucleic acids to design molecular nanostructures and nanoscaffolds that respond to specific biomarkers and, in doing so, can i) signal the presence of a tumor marker or ii) release...

PI: Massimo Olivucci

We propose to open a novel research line exploiting technologies shown to be effective for the automatic design and preparation of photo-responsive proteins. We plan to transfer/adapt such technologies to the design and preparation of photo-modulable RNA aptamers capable to optically control the translation at both the cellular and tissue...

PI: Laura Cerchia

Research activity: RNA aptamers, similarly to antibodies, form high-affinity bonds with their specific protein targets, thus representing an effective tool for active cancer targeting. However, being synthesized chemically, they are cheaper, non-immunogenic, more stable and reliable in terms of reproducibility compared to protein therapeutics. My group has a consolidated expertise...

PI: Geroloma Condorelli

Nucleic acid-based aptamers are oligonucleotides which, by virtue of their ability to fold into peculiar 3D architectures, recognize their targets with high affinity and specificity. This project aims to develop innovative nucleic acid-based aptamers as therapeutics and drug carriers in the form of aptamer-drug conjugates/complexes to enhance specificity and decrease...

PI: Roberto Improta

This project aims to develop suitable methods to track, control and, where required, enhance the delivery of RNA and RNA-targeting drugs in the different cellular compartments. In particular, by combining experimental and computational approaches, several ‘smart’ nanocarriers will be developed and tested for their ability to transport RNA, also labelled...

Task 6.2.3

Task Leader: Alfredo Nicosia
PI: Alfredo Nicosia, Salvatore Piano

PI: Salvatore Piano

The Unit of Internal Medicine and Hepatology of the University-Hospital of Padua deals with all diseases of internal interest, including disorders of the cognitive sphere, but in particular with liver diseases. The clinic is equipped for the diagnosis and treatment of all liver diseases, in particular viral hepatitis, liver cirrhosis...

PI: Alfredo Nicosia

In vivo transfer of genetic material represents the next generation technology platform for medicines targeting the cause of a genetic disease (Gene Therapy: GT) or exploiting the immune system to elicit a prophylactic or therapeutic response against infectious diseases and cancer (Genetic Vaccine: GV). In the frame of the WP6.3...