Things
Cascading call announced by the University of Modena and Reggio Emilia – Spoke 1 “Genetic Diseases” in compliance with the provisions of the art. 5 of Public Notice no. 3138 of 12/16/2021, as part of the PNRR funding - Mission 4, Component 2 "From research to business", Investment 1.4 "Strengthening research structures and creation of R&D "national champions", relating to the identified project with code CN00000041, entitled “National Center for Gene Therapy and Drugs based on RNA Technology”. The Call is aimed at public and private bodies strongly interested in introducing significant innovations in relation to products and processes and is aimed at expanding the research network established by the members of the National Center and financing new entities that can obtain results that integrate into the research program. research, expanding its technological skills and accelerating the process of designing and developing new treatments based on the Key Enabling Technologies covered by the CN RNA & Gene Therapy.
Who is it aimed at?
¿ Micro, Small and Medium Enterprises (MSMEs), external to the National Center and competing in single mode, having the dimensional parameters referred to in Annex I of REG (EC) n. 800/2008 of the Commission of 6 August 2008 (General block exemption regulation) in OJEU L 214 of 9.8.2008, including innovative start-ups; - Large Enterprises (GI), external to the National Center and competing in single mode ;- Public research bodies and institutions, including universities, and the bodies referred to in Legislative Decree no. 218/2016 (https://www.mur.gov.it/it/aree-tematiche/ricerca/il- research-system/public-research-institutions);- Scientific Hospitalization and Care Institutes – IRCCS, which are public or private in nature;- Bodies referred to in the Third Sector Code – Legislative Decree 3 July 2017 n.117 and subsequent amendments.
What does it predict
Through this call we intend to support research activities in the following areas: Track for Line of intervention A: Track A – Research Activities (RF, RI) Areas of development of Spoke A1 – DNA editing and RNA suppressors for the development of personalized therapies in Hemophilia A2 – Gene therapy with lentiviral vectors by transcriptional targeting at the endothelial level for the expression of FVIII in a tolerogenic manner. A3 – Advanced biological models for the evaluation and reduction of the immunogenicity of gene therapy vectors in hemophilia. A4 – Innovative methods of delivery of tissue-specific gene editing systems (CRISPR-Cas, base/prime editing) via metal or lipid nanoparticles in models of neurodegenerative diseases such as Friedreich's Ataxia. A5 – Design and validation of synthetic genes and molecules that modulate RNA transport, tissue targeting and bioavailability in hereditary neuromuscular diseases A7 – Research activities on non-liposomal, biocompatible and biomimetic nanocarriers for RNA/DNA delivery and tissue targeting and organs Track for Line of intervention B – South: Track A – Research Activities (RF, RI) Spoke development areas A6 – Development of methods for evaluating the safety of hematopoietic stem cell genome editing technologies for therapy gene of hereditary diseases A8 – Evaluation of the effects induced by an increased expression of mi R-486 on the secretion of myokines and on the myokine profile of circulating extracellular vesicles in mouse models of Central Core Disease. A9 – Identification of phylogenetically relevant molecules that modulate retinal metabolism in normal and pathological conditions and administration of the identified molecules to perturb and treat retinal disease models. A10 – Development and analysis of 3D neuromuscular cell models from the differentiation of patient PSCs. A11 – Analysis of epigenetic modifications in 2D and 3D cellular models investigated using ATACseq and Ch IPseq. A12 – Development of innovative methods for the in vitro production, cryopreservation and transfer of gametes and embryos from genetically modified livestock animal models for mitochondrial pathologies of nuclear origin Track B – Technology transfer – Proof of Concept (RI and SS) B1 – Optimization of delivery and third generation sequencing approach/small RNA seq single cell pipeline to treat retinal disease models
